American Society of Gene + Cell Therapy

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PROGRAM GUIDE
WALTER E. WASHINGTON
CONVENTION CENTER

25th Annual Meeting [Link]

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TABLE OF CONTENTS
A Welcome Letter from Beverly L. Davidson, Ph.D...........................................3
General Meeting Information
Mission + Vision.................................................................................................... 4
Abstract Publications............................................................................................ 4
Continuing Medical Education........................................................................... 4
Disclosure............................................................................................................... 4
Education Objectives........................................................................................... 5
Evaluation Method................................................................................................ 5
Needs....................................................................................................................... 5
Target Audience.................................................................................................... 5
Dates + Location................................................................................................... 6
Photography.......................................................................................................... 6
Printing Instructions............................................................................................. 6
Health + Safety.......................................................................................................7
On-site Safety Measures.................................................................................... 10
Registration...........................................................................................................11
Floorplans............................................................................................................. 12
Program Committee + Abstract Planning Committee...................................15
2022 ASGCT Program Committee.................................................................. 15
2022 Abstract Planning Committee................................................................ 15
2022 Annual Meeting Sponsors..........................................................................16
2022 Award Recipients.........................................................................................17
Annual Meeting Exhibitors...................................................................................19
Schedule at a Glance............................................................................................21
2022 Annual Meeting Schedule......................................................................... 39
Monday, May 16, 2022.......................................................................................40
Tuesday, May 17, 2022........................................................................................ 79
Wednesday, May 18, 2022.............................................................................. 106
Thursday, May 19, 2022................................................................................... 134
Abstract Reviewers............................................................................................. 150

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A WELCOME LETTER FROM BEVERLY L. DAVIDSON, PHD
Dear Colleagues,
On behalf of the American Society of Gene & Cell Therapy
(ASGCT), I am thrilled to welcome you to our first Annual
Meeting back in person since 2019. After almost three years
apart, I feel especially honored to join you and thousands
of our colleagues from around the world who believe in the
Society’s mission of advancing knowledge, awareness, and
education to expand the discovery and clinical application of
gene and cell therapies for alleviation of human disease.
Whether you’re gathering with us in person in Washington, D.C. or virtually, I hope
you’re looking forward to a week that will be packed with the latest research
developments in gene and cell therapy. Please remember that all attendees will
have on-demand access to the plenary lectures, oral abstract presentations,
scientific symposia, education sessions, virtual poster hall, and more, for 30 days
following the meeting.
I would like to extend a warm welcome to Drew Weissman, MD, PhD, and ASGCT
Past President Kathy High, MD, who will present the George Stamatoyannopoulos
Memorial Lecture on Wednesday morning and the Presidential Symposium on
Wednesday afternoon, respectively. We’ll also hear from former NIH Director
Francis Collins, MD, PhD, who will receive the first ASGCT Founders Award
recognizing his contributions to the field.
I’d like to congratulate our Annual Meeting award winners: Don Kohn, MD, recipient
of the Outstanding Achievement Award; Lindsey George, MD, Matthew Hirsch, PhD,
Morgan Maeder, PhD, and Christopher Peterson, PhD, recipients of the Outstanding
New Investigator Award; Kathy High, MD, recipient of the Jerry Mendell Award for
Translational Science; and P.J. Brooks, PhD, recipient of the Sonia Skarlatos Public
Service Award. Thank you for your important work to move the field forward.
If you’re here in person, make sure to visit the Exhibit Hall to learn about the latest
products and services from dozens of companies in the space. For those who are
on the job hunt or looking to build connections, our Career Fair and Mentor Meet-
Up are open to all meeting registrants.
Finally, I would like to thank all of you involved in the planning and execution of
the Annual Meeting, especially the Program Committee, Scientific and Education
Committees, abstract reviewers, staff, and all of our volunteers. I can’t think of a
better way to celebrate 25 years of our Society’s progress than to hold our first-
ever hybrid Annual Meeting for what we hope will be our largest audience to date.
Thank you for supporting ASGCT and enjoy the meeting!
Sincerely,
Beverly L. Davidson, PhD
President, ASGCT

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GENERAL MEETING INFORMATION
Mission + Vision
The mission of ASGCT is to advance knowledge, awareness, and
education leading to the discovery and clinical application of genetic and
cellular therapies to alleviate human disease. ASGCT’s strategic vision
is to be a catalyst for bringing together scientists, physicians, patient
advocates, and other stakeholders to transform the practice of medicine
by incorporating the use of genetic and cellular therapies to control and
cure human disease.

Abstract Publications
All abstracts accepted for presentation at the ASGCT 25th Annual Meeting
have been published in a May supplement of Molecular Therapy and are
searchable for free on [Link].

Continuing Medical Education

The ASGCT Board of Directors decided that the Society will not offer
CME credit for the 25th Annual Meeting.

Disclosure
In keeping with good practices, ASGCT requires faculty to disclose any
relevant financial interest or other relationship with the manufacturer(s) of
any commercial product(s) and/or provider(s) of commercial services that
are discussed in this educational activity.
Disclosures are published on the Annual Meeting website agenda and at
the beginning of every presentation.

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Educational Objectives
At the conclusion of the activity, the participant should be able to:
• Provide advice to patients who inquire about the potential of gene
and cell therapy or the availability of open clinical trials, based on their
exposure to the current clinical trials in gene and cell therapies.
• Better instruct their students in medical school and other health
venues using the state-of-the-art basic science and clinical trials data
presented at the meeting.
• Use the latest advances in gene and cell therapy to enhance their research
mission, as physician scientists conducting basic and clinical research.
• Demonstrate improved regulatory compliance in conducting gene
and cell therapy clinical trials, through exposure to National Institutes
of Health (NIH) and Food and Drug Administration (FDA) faculty during
the educational program.

Evaluation Method
An evaluation of the Annual Meeting is live and will be available for
30 days after the meeting’s conclusion. The evaluation will address
program content, presentation, and possible bias, and will be sent to all
attendees at the conclusion of the event.

Needs
Clinical gene transfer has become increasingly complex due to ongoing
developments in the fields of gene and cell therapy, together with
bioethics, research integrity, and financial conflicts, as well as federal
mandates, regulations, and guidelines. Oligonucleotide therapies, novel
vector development, host-vector interactions and vaccine therapies will
be discussed as well as many other scientific topics. This meeting will
provide an educational forum for scientists and clinicians to expand their
knowledge about the broad developments in these fields.

Target Audience
The target audience includes basic science and translational researchers,
clinical investigators, physicians, postdoctoral fellows, graduate students,
employees of federal government and regulatory agencies, patient
advocates, and other healthcare professionals with an interest in the
latest advancements in the fields of gene and cell therapy.

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Dates + Location
The ASGCT 25th Annual Meeting will be a hybrid event. The in-person
portion will be held at the Walter E. Washington Convention Center in
Washington, D.C. A virtual registration will also be offered. Both virtual
and in-person attendees will have access to the virtual platform and
on-demand content.
The meeting will begin on the morning of Monday, May 16, 2022 and
continue through 12 PM (ET) on Thursday, May 19, 2022. The Exhibit
Hall will be open daily 9:45 AM to 6:30 PM (ET) Monday, May 16 through
Wednesday, May 18 in Hall D. All ASGCT content and select sponsor-
generated content will be available to registrants on-demand for
30 days following the conclusion of the meeting.

Photography + Video Recording

ASGCT contracts with photographers and videographers throughout the
Annual Meeting to assist in the creation of future marketing materials. By
attending the 25th Annual Meeting you understand and agree that your
image may be recorded and used in ASGCT promotional materials.

Printing Instructions
To print this program:
1. Right click on the program and select “Print.”
2. In the print settings, select “Fit to printable area.”
3. Select “Print.”

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HEALTH AND SAFETY
Vaccine Requirement
The safety of meeting attendees is ASGCT’s top priority and we believe
the most effective way to help ensure that safety is for everyone to be
vaccinated against COVID-19. Please note that proof of vaccination
is required for all in-person meeting attendees prior to traveling
to Washington, D.C. 
We define “fully vaccinated” to be:
• At least 14 days past the final administration of the second dose in a
two-dose series (i.e. Pfizer or Moderna vaccines),
OR
• At least 14 days past a single-dose administration (i.e. Johnson &
Johnson vaccine)
ASGCT will accept a full course of immunization with any vaccine that has
been authorized by the World Health Organization (WHO) or the U.S. Food
and Drug Administration (FDA). The product may be either fully authorized
or authorized under an Emergency Use Authorization (EUA) or equivalent
at the time of travel. Vaccination with an agent that is not included on the
WHO or FDA website as authorized will not be accepted. 
Booster shots are not required but are recommended for any eligible
communities. For those unable to travel or meet the vaccination
requirement, ASGCT is offering a virtual registration option. 

Vaccine Verification
Prior to arriving on site, attendees will be asked to provide proof that
they are fully vaccinated with a vaccine approved by the WHO or FDA.
ASGCT is partnering with Safe Expo for vaccination verification services to
make your entry to the meeting faster, safer, and more secure. In-person
meeting attendees are required to upload your vaccination card to the
secure vaccination verification portal before the event. Please visit the
Safe Expo desk near registration for assistance with vaccine verification.

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COVID-19 Testing Available On-site
A negative COVID-19 test is not required to attend the meeting, but testing
is available for your convenience if you choose to utilize it. Onsite testing
will be available at the Annual Meeting during the hours below. ASGCT
is working with Safe Expo to manage the onsite health and safety plan.
If you want to test onsite, Safe Expo is offering three options. You will be
responsible for the cost of your test.
Rapid Antigen Test – $50 each
Rapid Molecular NAAT LAMP Test* – $150 each
Rapid RT-PCR – $200 each
*This test is different from a PCR, which requires a 24–48-hour
turnaround. This test is acceptable in Canada, Japan, Korea, and most of
Europe, but please double check your local health guidelines to be sure.

Testing Hours
COVID-19 testing is available in Salon D on the street level of the
convention center during the following hours. To save time, please
schedule your test in advance.
Sunday, May 15: 8:00 AM to 5:00 PM
Monday, May 16: 8:00 AM to 5:00 PM
Tuesday, May 17: 8:00 AM to 5:00 PM
Wednesday, May 18: 8:00 AM to 5:00 PM
If you test positive for COVID-19 at any point immediately before or during
the 25th Annual Meeting, you must surrender your badge and follow CDC
guidance on quarantine and isolation. You will not be allowed to attend
sessions in person until a negative test can be produced, although you will
still have access to the virtual platform. Information on individuals who
test positive, including their names, will be provided to staff overseeing the
COVID-19 testing and response efforts. Safe Expo will report positive test
results to the Washington D.C. Public Health Service as required by law.
The identity of individuals who test positive will be kept confidential to the
general public and meeting participants.

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International Attendees
International attendees should always check current travel restrictions
to the U.S. on the CDC website before planning travel arrangements. In
addition, please monitor regulations in your home country to ensure that
you will be able to return from the U.S.

Other Guidelines
Anyone experiencing flu-like symptoms or symptoms associated with
COVID-19 must refrain from attending the meeting in-person. The
CDC states the symptoms of COVID-19 include: fever or chills, cough,
shortness of breath or difficulty breathing, fatigue, muscle or body aches,
headaches, new loss of taste or smell, sore throat, congestion or
running nose, nausea or vomiting, diarrhea, or a fever (100.4º F or higher).
As a reminder, all registered attendees will have access to view the
event virtually.
If you are attending in person and you experience any
COVID-19 symptoms, please refrain from attending and notify 
healthandsafety@[Link] as soon as possible.

Please visit the Health & Safety section of the Annual Meeting website
for the most up-to-date information.

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ON-SITE SAFETY MEASURES
Masks
ASGCT is requiring its staff and all on-site attendees to wear masks
regardless of whether it is required by law at the time of the meeting.
Masks must be always covering both mouth and nose except when
actively eating or drinking, or when giving a presentation as an invited
program speaker. The mask requirement applies to the convention
center, hotels, shuttle buses, and anywhere an in-person ASGCT event
is being held. 
Disposable surgical masks will be available for free if you do not have a
proper facial covering.  

Physical Distancing
Meeting rooms will be set in theater style at their maximum capacity.
Attendees can choose to sit next to someone or distance themselves if
they prefer more personal space. All in-person attendees will have
access to the virtual platform to view sessions in real time if they feel
uncomfortable with the space in meeting rooms.  

Sanitization 
Even though current research shows that COVID-19 transmission is
unlikely to occur from touching surfaces, convention center staff will
regularly clean and disinfect surfaces to promote healthy sanitation. 
The convention center also has hand sanitizer locations which are stocked
and deployed strategically. They are monitored and replenished frequently. 

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REGISTRATION
East Salon
Sunday, May 15 | 7:00 AM–7:00 PM
Monday, May 16 | 7:00 AM–6:00 PM
Tuesday, May 17 | 7:00 AM–6:00 PM
Wednesday, May 18 | 7:00 AM–6:00 PM
Thursday, May 19 | 7:00 AM–12:00 PM

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 (LeveL 1)

Grand Lobby/Registration/Salons A–I

Meeting Rooms 101–103 & 140–160
Loading Dock entrance

Street Level

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RETAIL

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WALTER E. WASHINGTON CONVENTION CENTER

[Link]
 (LeveL 2)

exhibit Halls D & e

Meeting Rooms 201–210
east and West Overlook

Level Two

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GRAND LOBBY BRIDGE

[Link]
 Level Three
Meeting Rooms 301–306
Ballroom

Kitchen

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PROGRAM COMMITTEE + ABSTRACT PLANNING COMMITTEE
2022 ASGCT Program Committee
Thank you to the following individuals for serving on the ASGCT Program
Committee and helping with the overall development of the ASGCT
25th Annual Meeting.
CHAIR
Beverly L. Davidson, PhD, Children’s Hospital of Philadelphia
MEMBERS
Jeffrey Chamberlain, PhD, University of Washington
Helen Heslop, PhD, Baylor College of Medicine
Hans-Peter Kiem, MD, PhD, Fred Hutchinson Cancer Research Center
Maritza McIntyre, PhD, Advanced Therapies Partners
Carol Miao, PhD, Seattle Children’s Research Institute
Stephen J. Russell, MD, PhD, Mayo Clinic

2022 Abstract Planning Committee

Thank you to the following individuals for reviewing and selecting
abstracts for presentation in the Presidential Symposium and
Clinical Trials Symposium!
PRESIDENT
Beverly L. Davidson, PhD, Children’s Hospital of Philadelphia
ASGCT PRESIDENT-ELECT
Hans-Peter Kiem, MD, PhD, Fred Hutchinson Cancer Research Center
ASGCT SECRETARY + ABSTRACT CHAIR
Terence R. Flotte, MD, University of Massachusetts Medical School
MEMBERS
Jeffrey Chamberlain, PhD, University of Washington
Helen Heslop, PhD, Baylor College of Medicine

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 THANK YOU
25TH ANNUAL MEETING SPONSORS

Diamond

Gold

Silver

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AWARD RECIPIENTS
Outstanding Achievement Award
Donald B. Kohn, MD
Distinguished Professor, Department of Microbiology, Immunology & Molecular
Genetics, UCLA

Outstanding New Investigator Award

Lindsey George, MD
Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Matthew Hirsch, PhD
Associate Professor, Department of Ophthalmology, University of North Carolina
Morgan Maeder, PhD
Director, Payload Sciences, Chroma Medicine
Christopher Peterson, PhD
Staff Scientist, Clinical Research Division, Fred Hutchinson Cancer Research Center

The Outstanding New Investigator Award is supported by:

Jerry Mendell Award for Translational Science

Katherine High, MD
President of Therapeutics, AskBio

Sonia Skarlatos Public Service Award

Philip J. Brooks, PhD
Acting Director Office of Rare Diseases Research, National Center for Advancing
Translational Sciences, National Institutes of Health

Founders Award
Francis Collins, MD, PhD
Former Director, NIH

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Excellence in Research Awards
Excellence in Research Award winners will be announced during the
George Stamatoyannopolous lecture on Tuesday, May 17th.
The Excellence in Research Awards are supported by:

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EXHIBIT HALL DIRECTORY (as of 4/1/22)
The Exhibit Hall is located in Hall D, and will be open daily 9:45 AM to 6:30 PM (ET)
Monday, May 16 through Wednesday, May 18. Please view interactive floor plan here.

EXHIBITOR BOOTH NUMBER EXHIBITOR BOOTH NUMBER

10x Genomics 219 Comecer 244
908 Devices 541 Cook MyoSite 277
ABL Inc. 341 Corning, Inc. 536
Agilent Technologies 518 CPC 210
Akron Biotech 635 Curiox Biosystems 564
Aldevron 309 Cystic Fibrosis Foundation 665
Alfa Wassermann Separation Cytiva 609
Technologies 337 Cytotheryx 266
American Society for Transplantation Cytovance Biologics 426
and Cellular Therapy 128 Dyne Therapeutics 549
AmplifyBio 246 Eurofins BioPharma Product Testing 462
Andelyn Biosciences 148 Eurofins Viracor BioPharma 466
Anemocyte Srl 412 Evozyne 243
ArcticZymes Technologies 241 Exemplar Genetics 430
Avid Bioservices, Inc. 542 Flash Therapeutics 252
Avirmax, Inc. 619 Forecyte Bio USA Limited 365
Axion BioSystems 613 Forge Biologics 214
Azzur Group 520 FuGENE Transfection (Fugent LLC) 559
B Medical Systems 661 FUJIFILM Diosynth Biotechnologies 420
Baker 317 FUJIFILM WAKO CHEMICALS
Batavia Biosciences 643 U.S.A. CORPORATION 544
Beckman Coulter Life Sciences 170 Gator Bio 238
BioCentriq 144 G-CON Manufacturing 276
Biocut Systems 261 Gene Tools, LLC 510
Biognosys 233 Genezen Laboratories, Inc. 464
BioIVT 158 GenIbet 514
Biomere 571 GenoSafe 136
Bio-Rad Laboratories 245 GENSCRIPT USA INC. 280
BioSharingNetwork 215 GTS Scientific 126
Bio-Techne 452 Gyros Protein Technologies 557
Bristol Myers Squibb 543 Halo Labs 432
Catalent Cell & Gene Therapy 234 Informa Pharma Intelligence 114
Cellipont Bioservices 526 Innoforce US 118
CellVec Pte Ltd 562 Integrated DNA Technologies (IDT) 166
Celonic AG 235 InVitria 442
Center for Breakthrough Medicines 204 Iota Sciences 450
CEVEC Pharmaceuticals 363 KromaTiD 645
Charles River 369 L7 Informatics 272
Children’s Hospital of Philadelphia 565 Labcorp Drug Development
Clean Cells 164 (formerly Covance) 480
ClearPoint Neuro, Inc. 373

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EXHIBITOR BOOTH NUMBER EXHIBITOR BOOTH NUMBER
Leroy Biotech 359 ProtaGene 140
Lonza 570 Proteintech Group, Inc. 532
Lovelace Biomedical 558 PTC Therapeutics, Inc. 512
LUMICKS 217 Puresyn, Inc. 563
Malvern Panalytical 533 QIAGEN 516
Matica Biotechnology 142 Refeyn 357
MaxCyte 257 Renovate Biosciences 207
MaxQ Research 561 Repligen 203
MilliporeSigma 351 Resilience 160
Miltenyi Biotec 263 Rocket Pharmaceuticals 138
Mirus Bio 527 Sabai Global, and subsidiaries
MISSION BIO 548 of Clinical Biosafety Services,
Molecular Devices, LLC 275 Shield Consulting and Castle IRB 227
MYRIADE 339 Sarepta Therapeutics 428
NanoView Biosciences Ltd 529 SCIEX 615
National Organization for Serumwerk Bernburg AG 222
Rare Disorders (NORD) 116 SGS Vitrology Limited 361
Ncardia 226 SIRION Biotech 218
NECI 274 SKAN US, Inc. 470
NHLBI Gene Therapy STEMCELL Technologies 575
Resource Program (GTRP) 560 Synthego 262
NOF CORPORATION 221 TAAV 237
Northern Biomedical Research 476 Takara Bio USA 534
Novartis AG 122 Taysha Gene Therapies 472
NxGEN Vector Solutions, LLC 260 Terumo Blood and Cell Technologies 435
OBiO Technology 258 Texcell - North America, Inc. 146
Olympus 270 Thermo Fisher Scientific 345
Open Therapeutics 205 Touchlight DNA Services 212
OrganaBio 106 Ultragenyx Pharmaceutical Inc. 647
OriGen Biomedical 637 Unchained Labs 242
Oxford Biomedica (UK) Limited 240 Univercells Technologies 211
PackGene Biotech INC 627 VectorBuilder 178
Pall Corporation 504 Versiti 108
Penn Vector Core 551 Vertex Pharmaceuticals 313
PeproTech, Inc., VGXI, Inc. 319
part of Thermo Fisher Scientific 375 Vinta Bio, Inc. 528
PerkinElmer 220 Viralgen Vector Core 648
Pfizer 269 Virica Biotech 112
Pharmaron 631 Virovek Incorporation 546
PhenoVista Biosciences 617 VIVEbiotech 264
Polyplus-transfection 569 Wacker Biotech 254
Powered Research, LLC 134 Waisman Biomanufacturing 104
PPD, part of Thermo Fisher Scientific 349 WuXi Advanced Therapies 436
Precision For Medicine 566 Wyatt Technology 553
Precision NanoSystems Inc. 641 Yecuris 176
PROGEN 458 Yposkesi 448
Promega Corporation 547 Zymo Research Corp. 250

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022
7:00 AM–6:00 PM REGISTRATION
EAST SALON

9:45 AM–6:30 PM EXHIBIT HALL

HALL D
Connect with Exhibitors
Coffee Social | 9:45–10:15 AM
Coffee Social | 3:15–3:45 PM
Networking Reception & Poster Session I | 5:30–6:30 PM

8:00–9:45 AM EDUCATION SESSIONS

BALLROOM B
• Off-the-Shelf Cell Therapies—Beyond T-Cells
Co-chair: Blythe Sather, PhD Ballroom B
BALLROOM C
• AAV Vectors—From Basic Biology to Regulatory Hurdles
Chair: Erik Barton, PhD

8:00–9:45 AM SCIENTIFIC SYMPOSIA

ROOM 206
• Accelerated Approval for Gene Therapies
Co-chairs: Jeremy Allen and Jennifer Wellman
ROOM 207
• Function and Therapeutics Applications of Circular RNAs
(circRNAs)
Co-chairs: Mark Kay, PhD and Paloma Giangrande, PhD
ROOM 204
• Intersection Between Genetic Therapy and Society—
Nothing About Us Without Us
Chair: Maritza McIntyre, PhD
SALON G
• Translating Science Into Medicine: Moving From
Bench to Startup (Session 1 of 2)
Co-chairs: H. Trent Spencer, PhD and Nicole Paulk, PhD

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022
8:00–9:45 AM SCIENTIFIC SYMPOSIA
ROOM 102
• Preparing for Research Partnerships With
Patient Advocates
Co-chairs: Rachel Bailey, PhD and Jennifer Helfer, PhD
ROOM 201
• Stem Cells in Tissue Repair and Regeneration:
Insights From Model Systems
Co-chairs: TK and Sangeetha Vadakke-Madathil, PhD
BALLROOM A
• The Ultimate Personalized Gene and Cell Therapy for
Treatment of Cancer
Co-chairs: Daniela Bischof, PhD and Sunil Raikar, MD

Building tomorrow’s
cancer center.
Our goal is for every patient to get the best
treatment possible. We aim to provide treatment
that high-risk patients cannot get anywhere else.
We are:
• Expanding cancer cell therapy to target
solid tumors.
• Using αβ T-cell depleted haploidentical
stem cell transplants to improve
treatment options.
• Offering gene therapy trials for blood
and bone marrow failure disorders.

Learn more at [Link].

043372 | 03/2022
SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022
9:00–11:30 AM EXHIBITOR SHOWCASES
ROOM 209 | INDUSTRY INTERACTIONS
GenScript USA Inc. | 9:00–9:30 AM
10x Genomics | 10:00–10:30 AM
InVitria | 11:00–11:30 AM

10:15 AM–12:00 PM ORAL ABSTRACT SESSIONS

ROOM 204
• AAV Gene Therapy in Large Animal Models
Co-chairs: Heather Gray-Edwards, DVM, PhD
and Juliette Hordeaux, DVM, PhD
ROOM 201
• Biology of Gene Edited Cells
Co-chairs: Matthew Porteus, MD, PhD and Paula Rio, PhD
SALON H
• Oligonucleotide Therapeutics
Co-chairs: Shen Shen, PhD and Michelle Hastings, PhD
ROOM 206
• Cardiovascular and Pulmonary Diseases
Co-chairs: Margaret Sleeper, VMD and Uta Griesenbach, PhD
SALON G
• Musculo-skeletal Diseases
Co-chairs: Perry Shieh, MD, PhD and Lindsay Wallace, PhD
ROOM 102
• Immune Responses to AAV Vectors
Co-chairs: Julie Crudele, PhD and Ying Kai Chan, PhD
BALLROOM A
• Vector Manufacturing and Engineering I:
Deciphering AAV Vector Genomes
Co-chairs: H. Trent Spencer, PhD and John Gray, PhD

12:00–1:30 PM INDUSTRY SPONSORED SYMPOSIA

Labcorp Drug Development (formerly Covance) | Room 202
MaxCyte | Ballroom B
Sarepta Therapeutics | Ballroom C
Terumo Blood and Cell Technologies | Room 207

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022
12:00–1:00 PM STARTUP SHOWCASES
ROOM 209 | INDUSTRY INTERACTIONS
Chair: Li Ou, PhD

1:30–3:15 PM SCIENTIFIC SYMPOSIA

SALON H
• Competing With Best Available Care: Perspectives
on Lowering Burden of Treatment With Cell and
Gene Therapies
Co-chairs: Nimi Chhina, PhD and Kristin Van Goor, PhD
ROOM 102
• Cutting-edge Gene and Cell Therapy Research in Japan
Co-chairs: Noriyuki Kasahara, MD, PhD
and Takafumi Nakamura, PhD
BALLROOM A
• Immune Responses to Gene Therapy
Co-chairs: Allison Keeler, PhD and Melissa Rhodes, PhD
SALON G
• Translating Science Into Medicine: Moving From Bench
to Startup (Session 1 of 2)
Co-chairs: H. Trent Spencer, PhD
and Madhusudan Peshwa, PhD
ROOM 206
• Career Development Award Presentations

1:30–3:00 PM EXHIBITOR SHOWCASES

ROOM 209 | INDUSTRY INTERACTIONS
PerkinElmer | 1:30–2:00 PM
PTC Therapeutics, Inc. | 2:30–3:00 PM

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022
1:30–3:15 PM ORAL ABSTRACT SESSIONS
ROOM 201
• Gene and Cell Therapy Trials in Progress
Co-Chairs: Kevin Flanigan, MD and Barry Byrne, MD, PhD

3:30–6:00 PM TOOLS AND TECHNOLOGY FORUM I

ROOM 209 | INDUSTRY INTERACTIONS
Co-chairs: Afrooz Rashnonejad, PhD and Li Ou, PhD

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022
3:45–5:30 PM ORAL ABSTRACT SESSIONS
ROOM 204
• AAV Preclinical CNS Gene Therapy
Co-chairs: Allison Bradbury, PhD and Brian Bigger, PhD
BALLROOM A
• Gene Editing in Blood and Immune Disorders
Co-chairs: Maria-Grazia Roncarolo, MD and Jose Segovia, PhD
SALON H
• Synthetic Nanoparticle-Based Gene Transfer
Co-chairs: Hai-Quan Mao, PhD and Carol Miao, PhD
SALON G
• Tools and Approaches for Inborn Errors of Metabolism
Co-chairs: Gloria Gonzalez-Aseguinolaza, PhD
and Charles Venditti, MD, PhD
ROOM 102
• CAR T-cells and Beyond
Co-chairs: Chiara Bonini, MD and Avery Posey, PhD
ROOM 206
• Cancer - Oncolytic Viruses
Co-chairs: Paola Grandi, PhD, MBA and Marta Alonso, PhD
ROOM 207
• Cell-Based Cancer Immunotherapies I
Co-chairs: Barbra Sasu, PhD and Daniel Abate-Daga, PhD
ROOM 202
• Hematopoietic Stem Cell Gene Therapy
Co-chairs: Bernhard Gentner, MD and Anne Galy, PhD
ROOM 201
• Vector Manufacturing and Engineering II:
Next Generation Methods
Co-chairs: Kerry Dooriss, PhD and Johannes Van Der Loo, PhD

5:30–7:00 PM INDUSTRY SPONSORED SYMPOSIA

Pall Corporation | Ballroom B
Thermo Fisher Scientific - Patheon | Ballroom C

5:30–6:30 PM NETWORKING RECEPTION & POSTER SESSION I

EXHIBIT HALL | HALL D

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
TUESDAY, MAY 17, 2022
7:00 AM–6:00 PM REGISTRATION
EAST SALON

9:45 AM–6:30 PM EXHIBIT HALL

HALL D
Connect with Exhibitors
Coffee Social | 9:45–10:15 AM
Coffee Social | 3:15–3:45 PM
Networking Reception & Poster Session II | 5:30–6:30 PM

8:00–9:45 AM EDUCATION SESSIONS

ROOM 202
• Cancer Gene Therapy
Chair: Christopher LaRocca MD
BALLROOM C
• CRISPR/Cas9 Gene Editing—Concepts to In Vivo Editing
Chair: Nuria Morral, PhD
ROOM 207
• Integrating Retroviral Vectors
Chair: John Tisdale, MD

8:00–9:45 AM SCIENTIFIC SYMPOSIA

ROOM 102
• Gene- and Cell-based Therapies for Lung and GI
Chair: Alisha Gruntman, DVM, PhD
BALLROOM B
• Intended and Unintended Roles of Viral Vector
Heterogeneity in Gene Therapy
Co-chairs: Chen Ling, PhD and Brian Bigger, PhD
SALON G
• Musculoskeletal Gene Therapy: Progresses and Challenges
Co-chairs: Scott Q. Harper, PhD and Chunping Qiao, PhD
BALLROOM A
• Fireside Chat—State of the Field: Emerging
Regulatory Trends
Chair: Keith Wonnacott, PhD

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
TUESDAY, MAY 17, 2022
8:00–9:45 AM SCIENTIFIC SYMPOSIA
ROOM 204
• Symposium on Translating Tissue Engineering and
Regenerative Medicine Approaches Into Therapies
Co-chairs: Steven Becker, PhD and Lloyd Rose, PhD
ROOM 206
• The Ethical Gray Zone? Perspectives on the Development
and Governance of Gene and Cell Therapies for Human
Enhancement
Co-chairs: Eric Juengst, PhD and Benjamin Hurlbut, PhD
ROOM 201
• Therapeutic Applications of RNA Therapy Strategies
Co-chairs: Lauren E. Woodard, PhD and Loree Heller, PhD

8:00–9:45 AM LATE BREAKING ORAL ABSTRACT SESSIONS

SALON H

8:15–9:45 AM EXHIBITOR SHOWCASES

ROOM 209 | INDUSTRY INTERACTIONS
Bio-Rad Laboratories | 8:15–8:45 AM
Catalent Cell & Gene Therapy | 9:15–9:45 AM

10:15 AM–12:00 PM GEORGE STAMATOYANNOPOULOS

SPONSORED BY: MEMORIAL LECTURE AND AWARDS PRESENTATION
HALL E

12:00–1:30 PM INDUSTRY SPONSORED SYMPOSIA

Corning Life Sciences | Ballroom C
Cytiva | Ballroom B
Taysha Gene Therapies | Room 207
Vertex Pharmaceuticals Inc. | Room 202

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
TUESDAY, MAY 17, 2022
12:00–1:30 PM EXHIBITOR SHOWCASES
ROOM 209 | INDUSTRY INTERACTIONS
Bio-Techne | 12:00–12:30 PM
Precision for Medicine | 1:00-1:30 PM

1:30–3:00 PM OUTSTANDING NEW INVESTIGATOR SYMPOSIUM

SPONSORED BY: HALL E

3:00–5:00 PM NETWORKING EVENT—CAREER FAIR

SOUTH PRE-FUNCTION
Connect with employers and get a complimentary professional
headshot! Make sure you are registered for the Annual Meeting
to attend the Career Fair.

3:30–6:00 PM TOOLS AND TECHNOLOGY FORUM II

ROOM 209 | INDUSTRY INTERACTIONS
Co-chairs: Blythe Sather, PhD and Le Cong, PhD

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
TUESDAY, MAY 17, 2022
3:45–5:30 PM ORAL ABSTRACT SESSIONS
BALLROOM A
• Discoveries in Fundamental AAV Biology
Co-chairs: Nicole Paulk, PhD and Anna Maurer, PhD
SALON G
• Enhanced AAV Targeting
Chair: Ana Rita Batista, PhD
HALL E
• Gene Editing in Cancer and Complex Diseases
Co-chairs: Angelo Lombardo, PhD and Mara Pavel-Dinu, PhD
SALON H
• Physical Methods and Extracellular Vesicle-Based
Gene Transfer
Chair: Richard Heller, PhD
ROOM 201
• Inborn Errors of Metabolism Gene and Cell Therapies:
Proof-of-Concepts and Beyond
Co-chairs: Gerald Lipshutz, MD and Stephanie Cherqui, PhD
ROOM 204
• Applications of Improved Gene Therapy Methods in
Neurologic Disorders
Co-chairs: Kathrin Meyer, PhD and Steven Gray, PhD
ROOM 102
• Enhancing CAR-T Cell Efficacy
Co-chairs: Craig Sauter, MD and Barbara Savoldo, MD, PhD
ROOM 202
• Gene Therapy for Immunologic Diseases
Co-chairs: Donald Kohn, MD and Claire Booth, MBBS, PhD
ROOM 206
• Immune Responses to Gene Delivery and
Vaccine Approaches
Co-chairs: Matthew Gardner, PhD and Manish Muhuri, PhD

5:30–7:00 PM INDUSTRY SPONSORED SYMPOSIA

Anemocyte | Ballroom C
Charles River Laboratories | Room 207
QIAGEN LLC | Ballroom B

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
TUESDAY, MAY 17, 2022
5:30–6:30 PM NETWORKING RECEPTION & POSTER SESSION II
EXHIBIT HALL | HALL D

6:00–8:00 PM NETWORKING EVENT—NEW MEMBER MEETUP

ROOM 103
The New Member Mixer welcomes attendees who have joined
ASGCT over the past three years. Usually reserved for members
joining in the past 12 months, we’ve expanded the mixer to
include those who have joined since we were last in-person
in 2019. This informal setting will introduce new members
to the Society and highlight the many ways they can use our
resources as their professional hub. Attendees will also hear
from a longtime member who has adeptly used their Society
membership throughout their career.
SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
WEDNESDAY, MAY 18, 2022
7:00 AM–6:00 PM REGISTRATION
EAST SALON

9:45 AM–6:30 PM EXHIBIT HALL

HALL D
Connect with Exhibitors
Coffee Social | 9:45–10:15 AM
Coffee Social | 3:15–3:45 PM
Networking Reception & Poster Session III | 5:30–6:30 PM

8:00–9:45 AM EDUCATION SESSIONS

BALLROOM B
• Gene Therapy Beyond Cancer
Chair: Satiro De Oliveira, MD
ROOM 204
• Pre-clinical Models
Co-chairs: Erik Barton, PhD and Hildegard Buning, PhD

8:00–9:45 AM SCIENTIFIC SYMPOSIA

ROOM 206
• Combating Misinformation in Science
Co-chairs: Karen Bulaklak, PhD, Sarepta Therapeutics
and Edith Pfister, PhD, UMASS Chan Medical School
ROOM 102
• Challenges and Status in Development of Gene Therapies
in Low- and Middle-income Countries
Co-chairs: Richard Koya, MD, PhD and Savita Rangarajan, MD
BALLROOM A
• Gene Editing: New Technology Advances
Co-chairs: Thomas J. Cradick, PhD and Paula Rio, PhD
SALON G
• Inborn Metabolic Issues
Co-chairs: Anne Galy, PhD and Alessandro Aiuti, MD, PhD
ROOM 207
• Non-viral Delivery: A Diverse Toolbox Comes of Age
Co-chairs: Stefan Radtke, PhD and Chantal Pichon, PhD

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
WEDNESDAY, MAY 18, 2022
8:00–9:45 AM SCIENTIFIC SYMPOSIA
ROOM 201
• Overview of Cardiac Gene Therapy
Co-chairs: Jeffrey L. Ellsworth, PhD and Ryan L. Boudreau, PhD
ROOM 202
• Vector-associated Neural and Ocular Inflammation
Co-chairs: Christine N. Kay, MD and Paul A. Sieving, MD

8:00–9:45 AM LATE BREAKING ORAL ABSTRACT SESSIONS

BALLROOM C

8:15–9:45 AM EXHIBITOR SHOWCASES

ROOM 209 | INDUSTRY INTERACTIONS
Informa Pharma Intelligence | 8:15–8:45 AM
Curiox Biosystems | 9:15–9:45 AM

10:15–11:15 AM PLENARY SESSION

HALL E
The Outstanding Achievement Award Symposium
Chair: Beverly L. Davidson, PhD

11:15 AM–12:00 PM PLENARY SESSION

HALL E
Founder’s Award Presentation
Chair: Beverly L. Davidson, PhD

12:00–1:30 PM INDUSTRY SPONSORED SYMPOSIA

Dyno Therapeutics | Room 202
Miltenyi Biotec | Room 207
Thermo Fisher Scientific | Ballroom B

12:00–12:30 PM EXHIBITOR SHOWCASES

ROOM 209 | INDUSTRY INTERACTIONS
L7 Informatics | 12:00–12:30 PM

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
WEDNESDAY, MAY 18, 2022
1:30–3:15 PM PLENARY SESSION
SPONSORED BY: HALL E
Presidential Symposium and Presentation of Top Abstracts
Chair: Beverly L. Davidson, PhD

3:30–6:00 PM TOOLS AND TECHNOLOGY FORUM III

ROOM 209 | INDUSTRY INTERACTIONS
Co-chairs: Kay Whye Peng, PhD and Rachel Bailey, PhD

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
WEDNESDAY, MAY 18, 2022
3:45–5:30 PM ORAL ABSTRACT SESSIONS
SALON H
• RNA Virus Vectors
Co-chairs: John Tisdale, MD and Mario Amendola, PhD
BALLROOM A
• AAV Developments in Liver, T-cells, and Toxicity
Co-chairs: Nicole Paulk, PhD and Amanda Dudek, PhD
HALL E
• New Gene Editing Technologies and Applications
Co-chairs: Luca Biasco, PhD and Paula Cannon, PhD
ROOM 202
• Novel Therapeutic Targets to treat CNS Disorders
Co-chairs: Miguel Sena-Esteves, PhD
and Rebecca Ahrens-Nicklas, MD, PhD
ROOM 102
• Harnessing Innate Immunity for Cancer Immunotherapy
Co-chairs: Gianpietro Dotti, MD and Sarwish Rafiq, PhD
SALON G
• Cell-Based Cancer Immunotherapies II
Co-chairs: Masato Yamamoto, MD, PhD
and Irina Balyasnikova, PhD
ROOM 206
• Engineered Cell Therapies
Co-chairs: Bakhos Tannous, PhD
and M Graca Almeida-Porada, MD, PhD
ROOM 201
• Vector Manufacturing and Engineering 3:
Improving Vector Design and System Performance
Chair: Christopher Doering, PhD
ROOM 204
• Pharmacology/Toxicology Studies or Assay Development I
Co-chairs: Heikki Turunen, PhD and Eva Andres-Mateos, MD, PhD

5:30–6:30 PM NETWORKING RECEPTION & POSTER SESSION III

EXHIBIT HALL | HALL D

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
WEDNESDAY, MAY 18, 2022
8:00–11:00 PM NETWORKING EVENT—CLOSING NIGHT RECEPTION
INTERNATIONAL SPY MUSEUM
Join us for a night of fun during the Closing Night Reception
at Washington, D.C.’s International Spy Museum! Enjoy the
museum exhibits, light appetizers, and drinks.
You can purchase a ticket ($50 per person) for the reception
before or after you register for the meeting. Space is limited!

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
THURSDAY, MAY 19, 2022
7:00 AM–12:00 PM REGISTRATION
EAST SALON

8:00–9:45 AM SCIENTIFIC SYMPOSIA

ROOM 201
• Effective Regulatory Interactions
Co-chairs: Megan Zoschg-Canniere, PhD and Kit Shaw, PhD
BALLROOM B
• Emerging Clinical and Translational Safety Topics in
Cell and Gene Therapy
Co-chairs: Megan Hoban, PhD and TK
BALLROOM A
• Finding a “Cure”: The Promise and Perils of Gene Therapy,
In Utero and Beyond
Co-chairs: Randy J. Chandler, PhD
and Stephanie Cherqui, PhD
ROOM 207
• Global Experience With COVID-19 Vaccines
Co-chairs: Shan Lu, MD, PhD and David B. Weiner, PhD
SALON G
• Panel Discussion: What are the Most Pressing Issues
of the Future?
Co-chairs: TK and Rayne Rouce, MD
ROOM 204
• Cutting-edge Gene and Cell Therapy Research,
in Memory of Dr. Manfred Schmidt
Co-chairs: Juan A. Bueren, PhD and Hildegard Büning, PhD

8:00–9:45 AM ORAL ABSTRACT SESSIONS

BALLROOM C
• Clinical Trials Spotlight Symposium
Chair: Maria Grazia Roncarolo, MD

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SCHEDULE AT A GLANCE (ALL TIMES LISTED IN ET)
THURSDAY, MAY 19, 2022
10:15 AM–12:00 PM ORAL ABSTRACT SESSIONS
BALLROOM A
• Novel AAV Capsids for the Brain, Eye and Kidney
Co-chairs: Nicole Paulk, PhD and Andrew Steinsapir
BALLROOM C
• New Technologies for AAV Gene Therapy
Co-chairs: Phillip Tai, PhD and Aravind Asokan, PhD
BALLROOM B
• AAV Vectors - Clinical Studies
Co-chairs: Kathryn Wagner, MD, PhD and Christian Mueller, PhD
ROOM 202
• Delivery Technologies and In Vivo Gene Editing
Co-chairs: TJ Cradick, PhD and Daniela Cesana, PhD
ROOM 201
• Breakthroughs in Neuromuscular and Hearing Disorders
Co-chairs: Scott Q. Harper, PhD and Rachel M. Bailey, PhD
SALON G
• Ophthalmic and Auditory Diseases
Co-chairs: Hemant Khanna, PhD and Mariacarmela Allocca
ROOM 207
• Cell-Based Cancer Immunotherapies III
Co-chairs: Saad Kenderian and Michael Milone, MD, PhD
ROOM 204
• Cell Therapies for Hematological Disorders
Co-chairs: Andre Larochelle, MD, PhD
and Punam Malik, MD, PhD
ROOM 206
• Cell Therapy Product Engineering, Development
or Manufacturing
Co-chairs: Isabelle Riviere, PhD and Maksim Mamonkin, PhD
ROOM 102
• Pharmacology/Toxicology Studies or Assay Development II
Co-chairs: Angela Lynch, PhD and Eva Andres-Mateos, PhD

25th Annual Meeting [Link]

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 SCHEDULE
ALL TIMES LISTED IN ET
EDT

25th Annual Meeting [Link]

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

7:00 AM–6:00 PM REGISTRATION HOURS—EAST SALON

9:45 AM–6:30 PM EXHIBIT HALL OPEN—HALL D

9:45–10:15 AM Exhibit Hall Coffee Social
3:15–3:45 PM Exhibit Hall Coffee Social
5:30–6:30 PM Exhibit Hall Networking Reception
& Poster Session I

BALLROOM B
Off the Shelf Cell Therapies—Beyond T-cells
Chair: Blythe Sather, PhD, Tune Therapeutics
EDUCATION SESSIONS

8:00–8:24 AM Programming T-cell Therapies with CRISPR

Alexander Marson, MD, PhD, UCSF

8:24–8:48 AM The Next Generation of γδT Cell-based Therapies

Lawrence S. Lamb, Jr, PhD, IN8Bio

8:48–9:12 AM Epigenetic Programming to Enhance Cell Therapy

Charles A. Gersbach, PhD, Duke University

9:12–9:36 AM Progress in Pluripotent Cell Therapy Manufacturing:

A Personal 22-year Journey
Steve Oh, PhD, Bioprocessing Technology Institute, A*STAR

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

BALLROOM C
AAV Vectors—From Basic Biology to Regulatory Hurdles
Chair: Erik S. Barton, PhD, Pfizer, Inc.

8:00–8:20 AM How Comparative Studies of Mammalian- and

Insect-cell-derived AAV Can Enable Design of
Vector Manufacturing Platforms
Nicole Paulk, PhD, UCSF
EDUCATION SESSION

8:20–8:40 AM Understanding and Controlling Mechanisms of

AAV Immune-response-associated Toxicities
J. Fraser Wright, PhD, Stanford University School of Medicine

8:40–9:05 AM Harnessing AAV Membrane-associated Accessory Protein

to Improve Vector Supply and Quality
Kari J. Airenne, PhD, Kuopio Center for Gene and
Cell Therapy (KCT)

9:05–9:25 AM Understanding and Mitigating the Potential

for AAV-associated Oncogenesis
Denise Sabatino, PhD, Children’s Hospital of Philadelphia

9:25–9:45 AM Bridging AAV Biology to Platform and Process Innovation

Aravind Asokan, PhD, Duke University

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 206
Accelerated Approval for Gene Therapies
Co-chairs: Jeremy Allen, Spark Therapeutics and Jennifer Wellman, Akouos

8:00–8:15 AM Overview of the Accelerated Approval Pathway:

Definitions, Legislative History, and Purpose
Anna K. Abram, Akin Gump Strauss Hauer & Feld LLP

8:15–8:30 AM Patient Perspective: How Patients Weigh Benefit/Risk of

Treatment, and How to Weigh Unmet Need
SCIENTIFIC SYMPOSIA

Teonna Woolford, Sickle Cell Reproductive Health

Education Directive

8:30–8:45 AM Current Developments in Policy: Pathway Development

Consortium
Nina Hunter, PhD, REGENXBIO; Pathway Development
Consortium

8:45–9:00 AM Reimbursing Products After Accelerated Approval:

A Payer Perspective

9:00–9:15 AM The Significance of Accelerated Approval to Rare Disease

Drug Development
Emil Kakkis, MD, PhD, Ultragenyx

9:15–9:45 AM Panel Discussion

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 207
Function and Therapeutics Applications of Circular RNAs (circRNAs)
Co-chairs: Mark Kay, PhD, Stanford University School of Medicine
and Paloma Giangrande, PhD, Wave Life Sciences
SCIENTIFIC SYMPOSIA

8:00–8:35 AM Circular RNAs in Innate Immunity

Ling-Ling Chen, PhD, Shanghai Institute of Biochemistry
and Cell Biology

8:35–9:10 AM Circular RNA Immunology

Grace Chen, PhD, Yale University

9:10–9:45 AM In-situ CAR Therapy Using oRNA Lipid Nanoparticles

(LNPs) Regresses Tumors in Mice
Tom Barnes, PhD, Orna Therapeutics

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 204
Intersection Between Genetic Therapy and Society—
Nothing About Us Without Us
Chair: Maritza C. McIntyre, PhD, Advanced Therapies Partners

8:00–8:24 AM Does Where You Live Matter?

SCIENTIFIC SYMPOSIA

Michael Louella, University of Washington

8:24–8:48 AM Intersection Between Genetic Therapy and Society

Carla Elena Echeveste, All of Us

8:48–9:12 AM International Efforts to Expand Diversity of

Genomic Data to Improve Health Outcomes
Julie Makani, H3A Africa

9:12–9:36 AM Attracting and Retaining Diversity in Research

Danielle Quarles, Sana Biotechnology

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

SALON G
Translating Science Into Medicine: Moving From Bench to Startup
(Session 1 of 2)
Co-chairs: H. Trent Spencer, PhD, Emory University School of Medicine
and Nicole Paulk, PhD, UCSF

8:00–8:48 AM Translating Science into Medicine: Moving from

Bench to Startup
Deborah Palestrant, PhD, 5AM Ventures

8:48–9:36 AM How to Raise Seed/Series A Funding From Institutional

Biotech VCs for a CGT Startup
Timothy Miller, PhD, Forge Biologics
SCIENTIFIC SYMPOSIA

ROOM 102
Preparing for Research Partnerships With Patient Advocates
Co-chairs: Rachel M. Bailey, PhD, UT Southwestern Medical Center
and Jennifer Helfer, PhD, Encoded Therapeutics

8:00–8:24 AM Understanding the Patient Advocate’s Role

Florian Eichler, MD, Massachusetts General Hospital

8:24–8:48 AM Moving From Preclinical to Clinical Trials—

The Translational Path
Yael Weiss, MD, PhD, Mahzi Therapeutics

8:48–9:12 AM A Parent’s Journey Through Drug Development:

From Diagnosis to Preclinical Work to Clinical Trial
Allyson Berent, DVM, DACVIM, Foundation for Angelman
Syndrome Therapeutics; GeneTx Biotherapeutics; Angelman
Syndrome Biomarker and Outcome Measure Consortium

9:12–9:36 AM Lessons Learned From a Patient Advocate

Sharon King, Taylor’s Tale

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 201
Stem Cells in Tissue Repair and Regeneration: Insights From Model Systems
Chair: Sangeetha Vadakke-Madathil, PhD, Icahn School of Medicine at Mount Sinai

8:00–8:35 AM Interspecies Chimerism and Organogenesis

Jun Wu, PhD, UT Southwestern Medical Center

8:35–9:10 AM Sheep Model of Diseases Generated by CRISPR/Cas9

Irina Polejaeva, PhD, Utah State University

9:10–9:45 AM Engraftment of CD34 + Cells Gene-edited at the Sickle

Cell Disease Locus in Non-human Primate Models
John Tisdale, MD, National Institutes of Health;
SCIENTIFIC SYMPOSIA

National Heart, Lung, and Blood Institute

BALLROOM A
The Ultimate Personalized Gene and Cell Therapy for Treatment of Cancer
Co-chairs: Daniela Bischof, PhD, Indiana University School of Medicine
and Sunil S. Raikar, MD, Emory University

8:00–8:25 AM Strength/Limitation of Gene and Cell Therapy—

Route of Delivery
Richard Vile, PhD, Mayo Clinic

8:25–8:50 AM Cell Therapy Approaches with Genome-edited T-cells

Chiara Bonini, MD, Ospedale San Raffaele

8:50–9:15 AM Safety of Cell Therapy - CAR T-cells

New CAR Engineering
Gianpietro Dotti, MD, UNC School of Medicine

9:15–9:40 AM Personalizing Oncolytic Virotherapy and

Immunovirotherapy Approaches
Evanthia Galanis, MD, DSc, Mayo Clinic

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 VISIT US AT BOOTH 472
TO LEARN MORE.

WHO WE ARE Please join us for a

lunch symposium
Taysha Gene Therapies is on
a mission to eradicate
monogenic CNS disease. Accepting the Challenge:
We have combined our Innovative Approaches and Translational
team’s proven experience
in gene therapy drug Strategies in Gene Therapy Development
development with the
world-class UT Southwestern
Tuesday, May 17 from noon to 1:30 pm ET
Gene Therapy Program in Room 207
to build an extensive, fully
integrated AAV9 gene Complimentary lunch will be provided.
therapy pipeline with a goal
of dramatically improving Presented by Dr Suyash Prasad, Dr Steven Gray,
patients’ lives.
Dr Kimberly Goodspeed, and Dr Jagdeep Walia

Taysha Gene Therapies has a diverse pipeline

focused exclusively on monogenic disorders
of the central nervous system
Therapies in Development
2
GRT 1 shRNA

7
16 7 Neurodevelopmental
disorders 3 2

Neurodegenerative
diseases

miRNA 1 mini-gene

5 3 2
Genetic epilepsies

AAV, adeno-associated virus: CNS, central nervous system: GRT, Gene

Replacment Therapy: miRNA, microRNA: shRNA, short hairpin RNA.

© Taysha Gene Therapies 2022.

Contact us at medinfo@[Link]. All rights reserved.
[Link] TSHA-0168-2022
SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 209
Industry Interactions
GenScript USA Inc. | Fully Non-viral T cell Engineering With Hybrid
ssDNA Repair Templates

9:00–9:30 AM Brian Shy, MD, PhD

Associate Medical Director, UCSF
EXHIBITOR SHOWCASES

ROOM 209
Industry Interactions
10x Genomics | Case Study: Sequencing Strategies to Determine
Cellular States During Reprogramming and Regeneration
10:00–10:30 AM Peter Andersen, MS, PhD
Assistant Professor, The Johns Hopkins School of Medicine
Department of Medicine

ROOM 209
Industry Interactions
InVitria | Exhibitor Showcase

11:00–11:30 AM Presenter(s) to be announced

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 204
AAV Gene Therapy in Large Animal Models
Co-chairs: Heather Gray-Edwards, DVM, PhD, UMass Chan Medical School
and Juliette Hordeaux, DVM, PhD, University of Pennsylvania

10:15–10:30 AM 1: In vivo Selection of Randomly Integrated rAAV Vectors

Amita Tiyaboonchai, PhD, Oregon Health and
Science University
ORAL ABSTRACT SESSIONS

10:30–10:45 AM 2: Bicistronic AAV Gene Therapy for Tay-Sachs and

Sandhoff Diseases in the Sheep Model of Tay-Sachs
Toloo Taghian, PhD, UMass Chan Medical School

10:45–11:00 AM 3: Vagus Nerve Delivery of AAV9 to Treat Autonomic

Nervous System Dysfunction in Giant Axonal Neuropathy
Rachel Bailey, PhD, UT Southwestern Medical Center

11:00–11:15 AM 4: Assessment of Gene Therapy Treatment in the Pompe

Disease Canine Model
Megan Pope, University of Florida, Powell Gene
Therapy Center

11:15–11:30 AM 6: The Porcine Model for In Utero Gene Therapy

Apeksha Dave, MD, CHOP

11:30 AM–11:45 PM 7: Temporary Mechanical Support Improves Cardiac

AAV Gene Transfer Efficacy in a Pig HF Model
Renata Mazurek, MD, Mount Sinai

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 201
Biology of Gene Edited Cells
Co-chairs: Matthew Porteus, MD, PhD, Stanford University
and Paula Rio, PhD, CIEMAT

10:15–10:30 AM 8: Double Strand Break Free Genome Editing to Target

Hematopoietic Stem and Progenitor Cells: Therapeutic
Applicability in Fanconi Anemia
Laura Ugalde, CIEMAT/CIBERER/IIS-FJD

10:30–10:45 AM 9: Cellular Senescence and Inflammatory Programs are

Unintended Consequences of CRISPR-Cas9 Gene Editing
in Hematopoietic Stem and Progenitors Cells
Anastasia Conti, SR-TIGET

10:45–11:00 AM 10: The Choice of Template Delivery Mitigates the

ORAL ABSTRACT SESSIONS

Genotoxic Risk and Adverse Impact of Editing in Human

Hematopoietic Stem Cells
Aurelien Jacob, PhD, San Raffaele Telethon Institute for
Gene Therapy, IRCCS San Raffaele Scientific Institute

11:00–11:15 AM 11: Assessing Stealth and Sensed Base Editing in Human

Hematopoietic Stem/Progenitor Cells
Martina Fiumara, IRCCS San Raffele Hospital

11:15–11:30 AM 12: DNA Barcode as a Useful Tool to Study Hematopoietic

Stem Cell Fate in Gene Editing Strategies
Isabel Ojeda-Perez, PhD, Cell Technology Division,
Centro de Investigaciones Energéticas, Medioambientales
y Tecnológicas (CIEMAT) and Centro de Investigación
Biomédica en Red de Enfermedades Raras (CIBERER)

11:30–11:45 AM 13: High Frequency of AAV Integration at Double Strand

Breaks Induced in Preclinical Model of Gene Therapy and
in Edited Long-Term Engrafted HSPCs
Daniela Cesana, PhD, SR-TIGET

11:45 AM–12:00 PM 14: Base Editing of a γ-globin cis-regulatory Element in

Human Hematopoietic Stem Cells for Reactivation of
Therapeutic Fetal Hemoglobin
Panagiotis Antoniou, PhD, IMAGINE Institute, INSERM
UMR1163

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

SALON H
Oligonucleotide Therapeutics
Co-chairs: Shen Shen, PhD, Vertex Pharmaceuticals
and Michelle Hastings, PhD, Rosalind Franklin University

10:15–10:30 AM 15: Development of an AIMer for the Treatment of Alpha-1

Antitrypsin Deficiency
Prashant Monian, PhD, Wave Life Sciences

10:30–10:45 AM 16: STK-002, an Antisense Oligonucleotide (ASO) for the

Treatment of Autosomal Dominant Optic Atrophy (ADOA),
Localizes to Retinal Ganglion Cells (RGC) and Upregulates
OPA-1 Protein Expression After Intravitreal Administration
to Non-human Primates (NHP)
ORAL ABSTRACT SESSIONS

Aditya Venkatesh, PhD, Stoke Therapeutics

10:45–11:00 AM 17: Repeat Dosing with DYNE-101 is Well Tolerated and

Leads to a Sustained Reduction of DMPK RNA Expression
in Key Muscles for DM1 Pathology in hTfR1/DMSXL Mice
and NHPs
Stefano Zanotti, PhD, Dyne Therapeutics

11:00–11:15 AM 18: SNCA Reduction for the Treatment of

Synucleinopathie
Bradford Elmer, PhD, Sanofi

11:15–11:30 AM 19: Alpha-synuclein Lowering and Rescue of Motor

Phenotype by miRNA-based AAV Gene Therapy in In Vivo
Parkinson’s Disease Models
Seyda Açar Broekmans, PhD, uniQure Biopharma BV

11:30–11:45 AM 20: Discovery of Translation Initiation Elements Enabled

by a Parallel Arrayed Screen of Full-length Viral UTRs in
Synthetic Circular RNA
Alexander Wesselhoeft, Orna Therapeutics

11:45 AM–12:00 PM 21: Tumor-targeted miRNA Agent for

Pediatric Glioblastoma
Sunam Mander, PhD, University of Illinois at Chicago

25th Annual Meeting [Link]

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 206
Cardiovascular and Pulmonary Diseases
Co-chairs: Margaret Sleeper, VMD, University of Florida School of Veterinary
Medicine and Uta Griesenbach, PhD, Imperial College Faculty of Medicine

10:15–10:30 AM 22: Development of Passive Immunoprophylaxis

Against SARS-CoV-2 using Elderly and Immunodeficient
Mice Models
Yue Du, PhD, University of Oxford

10:30–10:45 AM 23: F/HN Pseudotyped Lentiviral Vector Uses Alpha 2,3

Sialylated N-Acetyllactosamine to Efficiently Transduce
Human Airway Cells
ORAL ABSTRACT SESSIONS

Rosie Munday, PhD, University of Oxford

10:45–11:00 AM 24: Extended Results from First-In-Human Clinical Trial

Of RP-A501 (AAV9:LAMP2B) Gene Therapy Treatment for
Danon Disease
Barry Greenberg, PhD, UC San Diego Medical Center

11:00–11:15 AM 25: Amphiphilic Peptides Deliver Adenine Base Editor

RNPs to Rhesus Monkey Airway Epithelial Cells In Vivo
Katarina Kulhankova, MD, PhD, University of Iowa

11:15–11:30 AM 26: Gene Therapy Induced Cholesterol Catabolism to

Treat Atherosclerosis and NASH
Mourad Toporsian, PhD, Repair Biotechnologies, Inc

11:30–11:45 AM 27: AAV Gene Therapy Using a Genetic Suppressor Treats

LMNA Dilated Cardiomyopathy in a Lmna Mouse Model
Following a Decline in Ejection Fraction
Yin Loon Lee, PhD, Nuevocor Pte Ltd

11:45 AM–12:00 PM 28: A First-in-Human Phase 1 Clinical Gene Therapy

Trial for the Treatment of Heart Failure Using a Novel
Re-Engineered Adeno-Associated Vector
Roger Hajjar, MD, Asklepios BioPharmaceutical, Inc.

25th Annual Meeting [Link]

52
SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

SALON G
Musculo-skeletal Diseases
Co-chairs: Perry Shieh, MD, PhD, UCLA
and Lindsay Wallace, PhD, Nationwide Children’s Research Institute

10:15–10:30 AM 29: Muscle-specific Tyrosine Kinase Chimeric

Autoantibody Receptor T-Cells (MuSK-CAART): A
Precision Cellular Immunotherapy for Antigen-specific B
Cell Depletion in MuSK Myasthenia Gravis
Sangwook Oh, PhD, University of Pennsylvania

10:30–10:45 AM 30: Novel Single AAV Vector Treatment for Congenital

Muscular Dystrophy Type 1A (MDC1A) Using CRISPR-
GNDM® Technology
Yuanbo Qin, PhD, Modalis Therapeutics Inc.
ORAL ABSTRACT SESSIONS

10:45–11:00 AM 31: Regeneration of Articular Cartilage, Suppression of

Synovial Inflammation, and Alleviation of Joint Pain After
Intra-articular Injection of ICM-203 in Canine OA Model
Minsun Park, Yonsei University

11:00–11:15 AM 32: Lentiviral Vector-Based Gene Therapy for Type II

Collagen Disorders
David Favre, PhD, Innoskel, SAS

11:15–11:30 AM 33: Multicenter AAV Gene Therapy Studies for SMARD1/

CMT2S Establish Safety and Efficacy in Multiple Animal
Models and Pave the Way for Initiation of a Phase I/II
Clinical Trial
Kathrin Meyer, PhD, Nationwide Childrens Hospital

11:30–11:45 AM 34: AAV-CRISPR-Cas13 Gene Therapy for FSHD:

DUX4 Gene Silencing Efficacy and Immune Responses to
Cas13b Protein
Afrooz Rashnonejad, PhD, The Abigale Wexner Research
Institute of Nationwide Children’s Hospital

11:45 AM–12:00 PM 35: Development of Dual AAV-mediated RNAi and Protein

Expression Therapy for Myotonic Dystrophy
Matthew Karolak, PhD, University of Washington

25th Annual Meeting [Link]

53
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MONDAY, MAY 16, 2022

ROOM 102
Immune Responses to AAV Vectors
Co-chairs: Julie Crudele, PhD, University of Washington
and Ying Kai Chan, PhD, Harvard University

10:15–10:30 AM 36: Characterizing AAV-mediated Immune Responses in

a Mouse Model of Duchenne Muscular Dystrophy
Melissa Spencer, UCLA

10:30–10:45 AM 37: Functional Assessment of T-cell Responses to AAV8

Empty Capsids in Healthy Volunteers
Holly Schroeder, PhD, AskBio
ORAL ABSTRACT SESSIONS

10:45–11:00 AM 38: Characterization of the Innate Immune Response to

AAV in Human Blood and the Central Role of Complement
Corinne Smith, PhD, Spark Therapeutics

11:00–11:15 AM 39: Deimmunized Micro-dystrophin Vectors Blunt Patient

Immunity in vitro & Restore Cardiac Functional Deficits
In Vivo in mdx4cv DMD Mice
Guy Odom, PhD, University of Washington

11:15–11:30 AM 40: Pretreatment With IVIG Reduces Peripheral

Transduction of AAV9 Delivered to the CNS
Cara West, Affinia Therapeutics, Inc.

11:30–11:45 AM 41: Differential T-cell Immune Responses to Deamidated

Adeno-associated Virus Vector
So Jin Bing, Food and Drug Administration

11:45 AM–12:00 PM 42: Interplay Between Plasmacytoid Dendritic Cells and

Kupffer Cells in IL-1R1-MyD88 Driven Cellular Immune
Responses to Hepatic AAV Gene Transfer
Sandeep Kumar, PhD, Indiana University

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

BALLROOM A
Vector Manufacturing and Engineering I:
Deciphering AAV Vector Genomes
Co-chairs: H. Trent Spencer, PhD, Emory University School of Medicine
and John Gray, PhD, Vertex Pharmaceuticals

10:15–10:30 AM 43: Direct ITR-To-ITR Nanopore Sequencing of Plasmid

and Vector Preparations and Their Implications for In Vivo
Gene-editing Vector Performance
Suk Namkung, ScM, UMass Chan Medical School

10:30–10:45 AM 44: Physico- and Biochemical Characterization of Linear

Covalently Closed dbDNATM for rAAV Manufacturing
ORAL ABSTRACT SESSIONS

Felix Bastida. DVM, PhD. TAAV Biomanufacturing

Solutions, S.L.

10:45–11:00 AM 45: Assessment and Comparison of Digital PCR Platforms

for AAV Viral Genome Titration
Stuart Nelson, MS, Prevail Therapeutics

11:00–11:15 AM 46: rAAV Vector Breakpoints Determined Using

Single-molecule, Modified Base Sequencing
Donald Selby, PhD, Homology Medicines

11:15–11:30 AM 47: Positioning Short-hairpin Stopper Sequences Outside

of the ITRs Reduces Encapsidation of Non-vector DNA in
rAAV Preparations
Mitchell Yip, UMass Chan Medical School

11:30–11:45 AM 48: Characterization of Residual DNA in rAAV Products

Made in the Baculovirus/Sf9 Platform
Daniel Barajas, PhD, BioMarin Pharmaceutical

11:45 AM–12:00 PM 49: NGS Based Evaluation of AAV Genome Integrity for
Improved Production and Function
Keith Connolly, Modalis Therapeutics

25th Annual Meeting [Link]

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 202
Labcorp Drug Development (formerly Covance) | Strategies and
Approaches to Optimize Your Non-clinical and Clinical Development
for Cell and Gene Therapies

12:00–1:30 PM Maryland Franklin, PhD

VP and Head, Enterprise Cell & Gene Therapy,
Labcorp Drug Development
Brian McIntosh, PhD
Lead, Cell & Gene Therapy/ Safety Assessment/Toxicology,
Labcorp Drug Development
Mark Cameron, MS
Director, Scientific Development, Preclinical Oncology,
INDUSTRY SPONSORED SYMPOSIA

Labcorp Drug Development

Darby Thomas, PhD
Director, Enterprise Cell & Gene Therapy,
Labcorp Drug Development
Alicia M. Baker McDowell, DRSc, MS
Head of Regulatory Strategy, Product Development &
Market Access Consulting, Labcorp Drug Development
Xcell Biosciences Representative
Xcell Biosciences
Akanksha Gupta, PhD
Executive Director, Enterprise Cell & Gene Therapy,
Labcorp Drug Development

Paul Byrne, MS
Associate Director, Enterprise Cell & Gene Therapy,
Labcorp Drug Development

Fred Derosier, DO
VP, Rare Diseases & Pediatrics and Clinical Lead, Enterprise,
Labcorp Drug Development
Ningchun Liu, PhD
Director, Enterprise Cell & Gene Therapy,
Labcorp Drug Development
TrakCel Representative
TrakCel

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

BALLROOM B
MaxCyte | Industry Sponsored Symposium

12:00–1:30 PM Presenter(s) to be Announced

INDUSTRY SPONSORED SYMPOSIA

BALLROOM C
Sarepta Therapeutics | Clinical Horizons for Investigational AAV-Based
Gene Transfer Therapy: The Rationale Behind rAAVrh74 as a Platform for
Neuromuscular Diseases
12:00–1:30 PM Presenter(s) to be Announced

ROOM 207
Terumo Blood and Cell Technologies | Balancing Scale, Risk and Cost:
How to Automate Your T cell Manufacturing Effectively

12:00–1:30 PM Stuart Gibb, PhD

Scientific Strategy Lead; Terumo Blood and
Cell Technologies

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

ROOM 209
Industry Interactions
Chair: Li Ou, PhD, Capsida Biotherapeutics

12:00–12:10 PM AviadoBio | A Revolution in Gene Therapy for

Neurodegenerative Disorders
Lisa Deschamps, MBA, CEO, AviadoBio

12:10–12:20 PM CellFE | Building the Future of Advanced Therapies

Through Microfluidics
STARTUP SHOWCASES

Alla Zamarayeva, PhD, CEO and Cofounder, CellFE

12:20–12:30 PM Nuevocor | Leveraging Mechanobiology to Treat

Genetic Cardiomyopathies
Yann Chong Tan, PhD, CEO, Nuevocor

12:30–12:40 PM Rejuvenation Technologies | Extending Telomeres to

Extend the Cellular and Human Lifespan
Colin Maraganore, Business Development Associate,
Rejuvenation Technologies

12:40–12:50 PM AAVnerGene Inc. | New Methods in AAV Production,

Characterization and Capsid Selection
Daozhan Yu, PhD, CEO, AAVnerGene Inc.

12:50–1:00 PM To be Announced

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

SALON H
Competing With Best Available Care: Perspectives on
Lowering Burden of Treatment With Cell and Gene Therapies
Co-chairs: Nimi Chhina, PhD, BioMarin
and Kristin Van Goor, PhD, Vertex Pharmaceuticals

1:30–1:45 PM Assessing Best Available Care and CGT:

A Disease-Agnostic Viewpoint
Craig Martin, Global Genes

1:45–2:00 PM FDA Considerations on Risk/Benefit for Best Available

SCIENTIFIC SYMPOSIA

Care Versus New Innovative Therapies

Peter Bross, MD, Food and Drug Administration

2:00–2:15 PM Assessing Best Available Care and Cell and

Gene Therapy in the Diabetes Context
Marjana Marinac, PharmD, Juvenile Diabetes
Research Foundation

2:15–2:30 PM Case Study of Zolgensma: Determining Labeling

Requirements for Gene Therapies to Guide Patient and
Physician Choice
Sitra Tauscher-Wisniewski, MD, Novartis Gene Therapies

2:30–2:45 PM Considerations for Gene Therapy Development in

Diseases with Available Therapies With Unmet
Medical Need
Jill Jarecki, PhD, BioMarin

2:45–3:15 PM Panel Discussion

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

ROOM 102
Cutting Edge Gene and Cell Therapy Research in Japan
Co-chairs: Noriyuki Kasahara, MD, PhD, UCSF
and Takafumi Nakamura, PhD, Tottori University School of Medicine

1:30–1:56 PM An RNA Gene Drug DVC1-0101 Based on Recombinant

Sendai Virus Vector to Treat Severe Intermittent
SCIENTIFIC SYMPOSIA

Claudication: Topline Results From Multicenter,

Double-blinded, Placebo-controlled Phase IIb
Clinical Trial
Yoshikazu Yonemitsu, MD, PhD, Kyushu University

1:56–2:22 PM Transposon-mediated CAR T-cells

Shigeki Yagyu, MD, PhD,
Kyoto Prefectural University of Medicine

2:22–2:48 PM Gene Therapy in a Mouse Model of OTC Deficiency With

Engineered AAV3 Vector
Kazuhiro Muramatsu, MD, PhD, Jinchi Medical University

2:48–3:14 PM Development of BBB-penetrating Heteroduplex

Oligonucleotides Regulating CNS Genes
Takanori Yokota, MD, PhD, Tokyo Medical and
Dental University

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

BALLROOM A
Immune Responses to Gene Therapy
Co-chairs: Allison M. Keeler, PhD, University of Massachusetts
Chan Medical School
and Melissa Rhodes, PhD, Kriya Therapeutics

1:30–1:55 PM Innate Immune Responses in Gene Engineering

Anna Kajaste-Rudnitski, PhD, San Raffaele Telethon Institute
for Gene Therapy

1:55–2:20 PM Systemic and Local Immune Responses to Intraocular

AAV Vector Administration
Deniz Dalkara, PhD, Institut de la Vision
SCIENTIFIC SYMPOSIA

2:20–2:45 PM Mechanisms, Monitoring, and Mitigation of Host Immune

Responses to AAV Gene Therapy Vectors
Kei Kishimoto, PhD, Selecta Biosciences

2:45–3:10 PM Drug-mediated Modulation of Immune Responses

to Gene Therapy
Klaudia Kuranda, PhD, Spark Therapeutics

SALON G
Translating Science Into Medicine: Moving From Bench to Startup
(Session 2 of 2)
Co-chairs: H. Trent Spencer, PhD, Emory University School of Medicine
and Madhusudan Peshwa, PhD, Tessera Therapeutics

1:30–2:18 PM Launching Innovation Into Gene Therapy Companies

Sheila Mikhail, JD, AskBio

2:18–3:06 PM Ex Vivo Gene-edited Cell Therapy Products

Jane Grogan, PhD, Graphite Bio

25th Annual Meeting [Link]

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 206
Career Development Award Presentations
Chair, Le Cong, PhD, Stanford University

1:35–1:55 PM Engineering Chimeric Gene Therapy Vectors with

Enhanced Packaging Capacity
Victoria Madigan, MIT
SCIENTIFIC SYMPOSIA

1:55–2:15 PM Therapeutic Isoform Specific Knockkdown Strategies for

Limb Girdle Muscular Dystrophy D1
Andrew Findlay, Washington University

2:15–2:35 PM Spatially-controlled Brain Gene Editing Guided by

Non-invasive Focused Ultrasound
Yeh-Hsing Lao, Columbia University

2:35–2:55 PM Polymeric Gene Delivery Nanoparticles to Treat

Multiple Sclerosis
Stephany Tzeng, John Hopkins University

2:55–3:15 PM Allo-iNKT Cells are Safe and Persist in

MHC-mismatched Dogs
Antonia Rotolo, University of Pennsylvania

25th Annual Meeting [Link]

62
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MONDAY, MAY 16, 2022

ROOM 209 | Industry Interactions

PerkinElmer | Exhibitor Showcase
EXHIBITOR SHOWCASES

1:30–2:00 PM Presenter(s) to be announced

ROOM 209 | Industry Interactions

PTC Therapeutics, Inc. | Pioneering In Gene Therapies:
An Update From PTC Therapeutics
2:30-3:00 PM Matthew B. Klein, MD, MS, FACS
Chief Operating Officer, PTC Therapeutics, Inc.
Philippe Moyen
Chief of Staff & Head of CMC Program Management –
Technical Operations, PTC Therapeutics, Inc.

High Quality Grade

Plasmid and
Minicircle DNA

GE scale!
Now in LAR
Starting material for
grafeting gmbh | visuals: [Link]

GMP production of mRNA,

viral vectors & CAR-T cells

25th Annual Meeting [Link]

63
[Link]
PlasmidFactory GmbH & Co. KG | Meisenstraße 96 | 33607 Bielefeld | Germany | info@[Link]
SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 201
Gene and Cell Therapy Trials in Progress
Co-chairs: Kevin Flanigan, MD, Nationwide Children’s Hospital
and Barry Byrne, MD, PhD, University of Florida

1:30–3:15 PM 50: High Anti-Sickling Potency of a Gamma Globin in

the Phase 1/2 MOMENTUM Study of ARU-1801 Gene
Therapy and Reduced Intensity Conditioning for Sickle
Cell Disease
Punam Malik, MD, Cincinnati Children’s Hospital
Medical Center

51: Safety and Outcomes of Intravenous scAAV9.U7-ACCA

for the Treatment of Duchenne Muscular Dystrophy
Caused by Exon 2 Duplications
ORAL ABSTRACT SESSIONS

Megan Waldrop, MD, Nationwide Children’s Hospital

52: RGX-121 Gene Therapy for the Treatment of Severe

Mucopolysaccharidosis Type II (MPS II): Interim Analysis
of Data from the First in Human Study
Roberto G Hiugliani, MD, Department of Genetics, UFRGS,
Medical Genetic Service, HCPA

53: Up to 10.5 Years of Follow-up in 17 Subjects Treated

With Hematopoietic Stem and Progenitor Cell Lentiviral
Gene Therapy for Wiskott-Aldrich Syndrome
Francesca Ferrua, IRCCS San Raffaele Scientific Institute  

54: Anti-GD2 CAR NKT Cells are Safe and Produce

Antitumor Responses in Patients with Relapsed/Refractory
Neuroblastoma
Andras Heczey, MD, Baylor College of Medicine

55: Exploratory Immuno-Safety Profile of EDIT-101,

a First-in-Human in vivo CRISPR Gene Editing Therapy
for CEP290-related Retinal Degeneration
Brian Duke, Editas Medicine, Inc.  

56: Hematopoietic Stem Cell Gene Therapy for Cystinosis:

Updated Results from a Phase 1/2 Clinical Trial
Stephanie Cherqui, PhD, UC San Diego  

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

ROOM 209
Industry Interactions
Co-chairs: Afrooz Rashononejad, PhD, The Abigail Wexner Research Institute
of Nationwide Children’s Hospital and Li Ou, PhD, Capsida Biotherapeutics

3:30–3:45 PM Forecyte Bio USA Limited | Accelerating Discovery to

Clinical with Trusted CDMO Services of Plasmids,
Viral Vectors and Cells
Shuyuan Zhang, CTO, Technology
3:45–4:00 PM Polyplus-transfection | Next-Generation Transfection
TOOLS AND TECHNOLOGY FORUM I

Reagent for Large Scale AAV Manufacturing

Mathieu Porte, R&D Bioproduction Manager,
Polyplus-transfection

4:00–4:15 PM STEMCELL Technologies | Generation of Large Numbers

of Megakaryocytes from Human Pluripotent Stem Cells
Leon Lin, PhD, Senior Scientist, RND, STEMCELL
Technologies

4:15–4:30 PM WuXi Advanced Therapies | TESSA Technology: Scalable,

Plasmid Free rAAV Manufacture
Ryan Cawood, Chief Scientific Officer, WuXi Advanced
Therapies
4:30–4:45 PM Pall Corporation | Quality by Design for Adeno-associated
Virus AAV Products
Peiqing Zhang, Strategic Technology Partnership Leader,
Pall Corporation

4:45–5:00 PM Molecular Devices | Next Generation Clone Screening

Workflows From Molecular Devices
Paula L. Feinberg-Zadek, PhD, BioPharma Field Applications
Scientist II, Molecular Devices

5:00–5:15 PM Cytiva | Purify Small Drug Volumes Better —

GMP Manufacturing of Viral Vectors
Fredrik Lundström, Senior Product Manager Downstream
Hardware Single-use Bioprocess, Cytiva

25th Annual Meeting [Link]

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TOOLS AND TECHNOLOGY FORUM I

5:15–5:30 PM MilliporeSigma | Bioprocess Development Using the

VirusExpress® 293 AAV Platform
Eva Fong Principal Scientist, Virus and Gene Therapy
Bioprocessing, MilliporeSigma
5:30–5:45 PM Dyne Therapeutics | FORCETM Platform for Targeted
Delivery of Oligonucleotide Therapeutics in Muscle
Diseases
Ashish Dugar, Senior VP, Global Head of Medical Affairs,
Dyne Therapeutics
5:45–6:00 PM Andelyn Biosciences | Reliable Scale-Up to 2000L
in Gene Therapy Manufacturing
Wade Macedone, Chief Operations Officer,
Andelyn Biosciences

25th Annual Meeting [Link]

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SCHEDULE (ALL TIMES LISTED IN ET)
MONDAY, MAY 16, 2022

ROOM 204
AAV Preclinical CNS Gene Therapy
Co-chairs: Allison Bradbury, PhD, Nationwide Children’s Hospital
and Brian Bigger, PhD, University of Manchester

3:45–4:00 PM 57: Selection of Clinical Doses for SBT101, an

AAV9-hABCD1 Vector for the Treatment of
Adrenomyeloneuropathy
D.W. Anderson, SwanBio Therapeutics Ltd

4:00–4:15 PM 58: AAV-ARSA Mediated Gene Replacement for the

Treatment of Metachromatic Leukodystrophy
ORAL ABSTRACT SESSIONS

Shyam Ramachandran, PhD, Sanofi

4:15–4:30 PM 59: Three Examples of Long-term AIDS-virus Suppression

Using AAV-Delivered Monoclonal Antibodies
Jose Martinez-Navio, Miller School of Medicine,
University of Miami

4:30–4:45 PM 60: AAV-mediated Delivery of Anti-HIV Antibodies to the CNS

Jose Martinez-Navio, Miller School of Medicine,
University of Miami

4:45–5:00 PM 61: Antibody Gene Therapy for Rabies Encephalitis

Amanda Gross, PhD, Auburn University

5:00–5:15 PM 62: Development of an Intrathecal AAV9/AP4M1 Gene

Therapy for Hereditary Spastic Paraplegia 50 (SPG50)
Xin Chen, UT Southwestern Medical Center

5:15–5:30 PM 63: CNS Penetrant AAV Vectors Encoding HER2 Antibodies

Reduce Tumor Burden in Models of Breast Cancer
Brain Metastasis
Dan Laks, PhD, Voyager Therapeutics

25th Annual Meeting [Link]

67
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MONDAY, MAY 16, 2022

BALLROOM A
Gene Editing in Blood and Immune Disorders
Co-chairs: Maria-Grazia Roncarolo, MD, Stanford University
and Jose Segovia, PhD, CIEMAT

3:45–4:00 PM 64: Two is Better Than One: CRISPR/Cas9 Based Gene

Editing with FOXP3 Isoforms for IPEX Therapy
Esmond Lee, PhD, Stanford

4:00–4:15 PM 65: Preclinical Safety and Feasibility Study of a

CRISPR/Cas9 Gene Editing Platform to Treat Wiskott
Aldrich Syndrome
Alessia Cavazza, PhD, UCL Institute of Child Health

4:15–4:30 PM 66: Development of a Beta-globin Gene Replacement

ORAL ABSTRACT SESSIONS

Strategy as a Therapeutic Approach for β-Thalassemia

Beeke Wienert, PhD, Graphite Bio, Inc.

4:30–4:45 PM 67: Adenine Base Editor-mediated Correction of

Three Prevalent and Severe β-thalassemia Mutations
Giulia Hardouin, Imagine Institute, INSERM UMR1163

4:45–5:00 PM 68: Improvement of PKLR-gene Editing in Human

Hematopoietic Stem and Progenitor Cells Towards its
Clinical Application for Pyruvate Kinase Deficiency
Isabel Ojeda-Pérez, PhD, Cell Technology Division,
Centro de Investigaciones Energéticas, Medioambientales
y Tecnológicas (CIEMAT) and Centro de Investigación
Biomédica en Red de Enfermedades Raras (CIBERER)

5:00–5:15 PM 69: Site-specific Editing Methods to Reverse Severe

Combined Immunodeficiency (SCID) in Athabascan-
speaking Native Populations
Patricia Claudio Vázquez, University of Minnesota

5:15–5:30 PM 70: Development of a Double shmiR Lentivirus

Effectively Targeting Both BCL11A and ZNF410 for
Enhanced Induction of Fetal Hemoglobin to Treat
β-hemoglobinopathies
Boya Liu, Boston Children’s Hospital

25th Annual Meeting [Link]

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MONDAY, MAY 16, 2022

SALON H
Synthetic Nanoparticle-Based Gene Transfer
Co-chairs: Hai-Quan Mao, PhD, Johns Hopkins University
and Carol Miao, PhD, Seattle Children’s Research Institute

3:45–4:00 PM 71: Building a Genetic Medicine Platform for

DNA-encoded Antibody Therapeutics
Kevin Hollevoet, PhD, PharmAbs - KU Leuven University

4:00–4:15 PM 72: Mechanisms of siRNA Delivery by Cyclic

Amphipathic Peptides
ORAL ABSTRACT SESSIONS

Uday Baliga, BS, MS, University of Rochester

4:15–4:30 PM 73: In Vivo Spatially Targeted Nonviral Optical Delivery

of Genes in Mice to NHPs
Sanghoon Kim, PhD

4:30–4:45 PM 74: Particle Size Engineering to Enhance mRNA Delivery

Efficiency via Biodegradable Carriers In Vivo
Yizong Hu, Johns Hopkins University

4:45–5:00 PM 75: Delivery of CRISPR/Cas9 mRNA LNPs to Repair a

Small Deletion in FVIII Gene in Hemophilia A Mice
Chun-Yu Chen, PhD, Seattle Children’s Research Institute

5:00–5:15 PM 76: Ministring DNA: A Durable and Safe Non-viral

Delivery Platform
Ting-Yen Chao, Seattle Children’s Research Institute

5:15–5:30 PM 77: Nano-structures for Efficient and Transgene-free

Immune Cell Transfection
Andy Tay, PhD, National University of Singapore (NUS)

25th Annual Meeting [Link]

69
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MONDAY, MAY 16, 2022

Salon G
Tools and Approaches for Inborn Errors of Metabolism
Co-chairs: Gloria Gonzalez-Aseguinolaza, PhD, Vivet Therapeutics
and Charles Venditti, MD, PhD, NIH

3:45–4:00 PM 78: Nuclease-Free Promoterless Genome Editing for

Wilson Disease
Agnese Padula, Telethon Institute of Genetics
and Medicine (TIGEM)

4:00–4:15 PM 79: Systemic Gene Therapy Using the Novel

Adeno-Associated Viral Vector 44.9
Randy Chandler, PhD, NHGRI, NIH
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 80: Single, Systemic Administration of BEAM-301

Mitigates Fasting Hypoglycemia and Restores Metabolic
Function in a Transgenic Mouse Model of Glycogen
Storage Disease Type Ia
Yvonne Aratyn-Schaus, PhD, Beam Therapeutics

4:30–4:45 PM 81: CRISPR-mediated Insertion of a Targeted GAA

Transgene into Hepatocytes Provides Effective,
Long-lasting Gene Therapy in Neonate and Adult Pompe
Disease Mice
Andrew Baik, MD, Regeneron Pharmaceuticals

4:45–5:00 PM 82: A Novel Human Liver Chimeric Mouse Model Lacking

the Murine AAVR Gene for Validation of AAV Gene
Therapy Vectors
Tong Chen, MD, PhD, Duke University

5:00–5:15 PM 83: Lentiviral-Based Genetic Correction of IL10RB-Defect

to Treat Very Early Onset-Inflammatory Bowel Disease
Adele Mucci, Dana Farber/Boston Children’s Cancer and
Blood Disorders Center, Harvard Medical School

5:15–5:30 PM 84: Ex vivo Editing of Hematopoietic Stem Cells for

Erythroid Expression of Therapeutic Proteins In Vivo for
LAL-D Therapy
Marine Laurent, Genethon

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ROOM 102
CAR T-cells and Beyond
Co-chairs: Chiara Bonini, MD, Università Vita Salute San Raffaele
and Avery Posey, PhD, University of Pennsylvania

3:45–4:00 PM 85: A Genome-Scale Screen for Synthetic Drivers of

T-cell Proliferation
Mateusz Legut, PhD, NY Genome Center

4:00–4:15 PM 86: CD45RA Expressing PBMCs Effect the Outgrowth of

Epstein-Barr Virus Antigen Specific T-cells
Sandhya Sharma, Baylor College of Medicine
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 87: IFNg Impedes Antigen-Specific Proliferation of CAR-T

with a CD28, but not 4-1BB, Costimulatory Domain
Stefanie Bailey, PhD, Massachusetts General Hospital

4:30–4:45 PM 88: Naturally Occurring CD7- T-cells Mark a Functional

Effector and Persistent CAR T-cell Population
Jaquelyn Zoine, PhD, St. Jude Children’s Research Hospital

4:45–5:00 PM 89: Tim-4-Chimeric Engulfment Receptor (CER) T-cell

Therapy Elicits Phosphatidylserine-Dependent Cytotoxic
and Antigen-Presenting Cell-Like Function and Synergizes
with Approved BTK Inhibitors for the Treatment of
Hematologic Malignancies
Daniel Corey, MD, CERo Therapeutics

5:00–5:15 PM 90: Peptide-scFv Bispecific CAR T-cells targeting Acute

Myeloid Leukemia
Jaquelyn Zoine, PhD, St Jude Children’s Research Hospital

5:15–5:30 PM 91: CD28-based B7-H3 CAR T-cells Have Superior

Anti-glioma Efficacy in an Immune Competent Glioma
Model While Suppressive Macrophages at Tumor Edges
are Associated with Therapeutic Resistance
Dalia Haydar, PharmD, PhD, St Jude Children’s
Research Hospital

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ROOM 206
Cancer — Oncolytic Viruses
Co-chairs: Paola Grandi, PhD, MBA , CG Oncology
and Marta Alonso, PhD, University Hospital of Navarra

3:45–4:00 PM 92: The Potential of Oncolytic Virotherapy in

Synovial Sarcoma
Steven Robinson, Mayo Clinic

4:00–4:15 PM 93: Oncolytic Adenovirus Expressing IFN-alpha with

Chemoradiation (GEM+nab-PTX) Synergistically Inhibits
Pancreatic Cancer Cell Growth In Vitro and
In Vivo
ORAL ABSTRACT SESSIONS

Shuhei Shinoda, MD, PhD, University of Minnesota

4:15–4:30 PM 94: Oncolytic HSV-1 rQNestin34.5v2 Sensitizes

IDH1-mutated Glioma to Immunotherapy
Eleni Panagioti, PhD, Brigham and Womens Hospital,
Harvard Medical School

4:30–4:45 PM 95: Characterization of the Cancer-Targeted Oncolytic

Adenoviruses with Fiber-Knob Modification
Mizuho Sato-Dahlman, PhD, University of Minnesota

4:45–5:00 PM 96: Optimizing NIS-expression for Oncolytic

Adenovirus-based Radiotherapy and Imaging of
Breast Cancer
Robert Sacha, PhD, University of Minnesota

5:00–5:15 PM 97: Tumor Regression of Oncolytic Adenovirus-treated

Melanoma Rely on the Gut Microbiome
Lorella Tripodi, PhD, CEINGE Biotecnologie Avanzate

5:15–5:30 PM 98: Oncolytic Adenovirus with Hyaluronidase

Activity that Evades Neutralizing Antibodies and
Allows Re-administration: VCN-11
Ramon Alemany, PhD, VCN Biosciences

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ROOM 207
Cell-based Cancer Immunotherapies I
Co-chairs: Barbra Sasu, PhD, Allogene
and Daniel Abate-Daga, PhD, H. Lee Moffitt Cancer Center and Research Institute

3:45–4:00 PM 99: Endowing Universal CAR T-cell With

Immune-Evasive Properties Using TALEN-Gene Editing
Julien Valton, PhD, Cellectis INC

4:00–4:15 PM 100: Engineering Stealth CAR T-cells to Evade the Host

Immune Responses
Korneel Grauwet, PhD/Ir, Massachusetts General Hospital /
Harvard Medical School
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 101: Tumor-directed, Myeloid Cell-based Cytokine

Gene Delivery Unleashes CAR T Cells in the
Immunosuppressive Glioblastoma Microenvironment
to Control Tumor Growth
Federico Rossari, MD

4:30–4:45 PM 102: Activation Regulated Gene Circuit for Controlling

Payload Expression in Cell Therapies
Michelle Hung, PhD, Senti Biosciences

4:45–5:00 PM 103: Preclinical Development of Safe and Effective

T-Cell Receptors Specific for Mutant KRAS G12V and
G12D Peptides
Tijana Martinov, PhD, Fred Hutchinson Cancer
Research Center

5:00–5:15 PM 104: Disruption of H3K9me3-mediated Gene Silencing

Augments CAR T-cell Functional Persistence
Nayan Jain, Memorial Sloan Kettering Cancer Center

5:15–5:30 PM 105: Mechanisms Regulating the Resistance of Normal

T-Cells to CD5 CAR-mediated Cytotoxicity
Royce Ma, Baylor College of Medicine

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ROOM 202
Hematopoietic Stem Cell Gene Therapy
Co-chairs: Bernhard Gentner, MD, SR-TIGET and Anne Galy, PhD, GENETHON

3:45–4:00 PM 106: Inhibition of P38-MAPK Counteracts Culture Stress

Induced by e x vivo Expansion of Hematopoietic Stem and
Progenitor Cells (HSPCs) for Efficient Genetic Engineering
Lucrezia Della Volpe, PhD, San Raffaele Telethon Institute for
Gene Therapy

4:00–4:15 PM 107: Targeting CX3CR1 Gene to Improve Microglia

Reconstitution and Transgene Delivery into the CNS Upon
Hematopoietic Stem and Progenitor Cell Transplant
Annita Montepeloso PhD, Dana-Farber/ Boston Children’s
Cancer and Blood Disorders
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 108: Ex vivo Lentiviral-mediated Gene Therapy for

Patients with Fanconi Anemia [Group A]: Updated Results
From Global RP-L102 Clinical Trials
A Czechowicz, MD, PhD, Stanford University School of
Medicine and Lucile Packard Children’s Hospital

4:30–4:45 PM 109: Preclinical Evidences Towards Lentiviral Gene

Therapy for RPS19-Diamond Blackfan Anemia Patients
Susana Navarro, PhD, CIEMAT/CIBERER/IIS-FJD

4:45–5:00 PM 110: A Mouse Model of Severe Alpha-Thalassemia

With Abnormal Iron Metabolism, Erythropoiesis and
Coagulation can be Rescued by a Novel Gene
Therapy Approach
Maxwell Chappell, University of Pennsylvania

5:00–5:15 PM 111: Treating Sickle Cell Disease With Lentiviral Vectors

Combining an Anti-Sickling βAS3-globin Gene with BCL11A
and ZNF410 MicroRNA Adapted Short Hairpin RNAs
Kevyn Hart, University of California, Los Angeles

5:15–5:30 PM 112: Transplantation Without Myeloablation: Novel

Conditioning Enables Robust Repopulation of Macrophage/
microglia Niches by Bone Marrow-derived Cells
Natalia Gomez-Ospina, MD, PhD, Stanford

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ROOM 201
Vector Manufacturing and Engineering II: Next Generation Methods
Co-chairs: Kerry Dooriss, PhD, City of Hope
and Johannes Van Der Loo, PhD, Children’s Hospital of Philadelphia

3:45–4:00 PM 113: Stable BaEVRless Producer Cell Line for the

Production and In Vivo Application of Alpha-Retroviral
Particles
Denise Klatt, PhD, Dana Farber Cancer Institute, Harvard
Medical School

4:00–4:15 PM 114: Generation and Characterization of Rep Expressing

AAV Packaging Cell Lines
Lovro Jalšić, Université Laval
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 115: Synthetic Biology Approach to Nucleic Acid

Clearance in Lentiviral Vector Production
Sadfer Ali, EngD, University College London

4:30–4:45 PM 116: Modification and Optimization of an AAV Purification

Process to Accommodate Increased Upstream Yield and
Reduce Manufacturing Bottlenecks
Nick DiGioia, LogicBio Therapeutics Inc

4:45–5:00 PM 117: Adenovirus Purification Method Using Scalable

System With Single Use Anion Exchange Fiber
Chromatography Capsule
Masato Yamamoto, MD, PhD, University of Minnesota

5:00–5:15 PM 118: Membrane-Associated Accessory Protein Variants

Improve Adeno-Associated Virus Production in
HEK293 Cells
Adam Schieferecke, MS, University of California, Berkeley

5:15–5:30 PM 119: AAV Manufacturing with Stable Helper-virus

Free ELEVECTA® Producer Cells for Industrial Scale
Vector Production
Ines do Carmo Gil Goncalves, PhD, CEVEC Pharmaceuticals
GmbH

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BALLROOM B
Pall Corporation | Successful Gene Therapy Scale-up, Start-up
and Facility Expansion
5:30–7:00 PM Clive Glover, PhD
General Manager, Gene Therapy, Pall Corporation

REGENXBIO Representative
REGENXBIO
INDUSTRY SPONSORED SYMPOSIA

Emily Moran
VP of Vector Manufacturing, Center for Breakthrough
Medicine

Avi Nandi
VP Process Development, Center for Breakthrough Medicine

BALLROOM C
Thermo Fisher Scientific-Patheon | Integrating Development and
Manufacturing Services to Bring Viral Vector Products to Market

5:30–7:00 PM Kim Watanabe, PhD

General Manager/Site Head, Translational Services,
Thermo Fisher Scientific

Akanksha Nagpal, PhD

Sr Director, Bioprocess Development,
Thermo Fisher Scientific

Christopher Murphy
Vice President/General Manager, Viral Vector Services,
Thermo Fisher Scientific

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HALL D
Networking Reception & Poster Session I
5:30–6:30 PM

ENGINEERING
HOPE
Discover how we’re using gene therapies to help build
a better future for patients with serious diseases.

[Link]

© 2022 MeiraGTx. All Rights Reserved.

25th Annual Meeting [Link]
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 GOLD SPONSOR

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THE STATE OF
ADVANCED GENETIC
THERAPIES IN 2022
Wednesday, May 18 | 8:15am - 8:45am (EST)

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SCHEDULE (ALL TIMES LISTED IN ET)
TUESDAY, MAY 17, 2022

7:00 AM–6:00 PM REGISTRATION HOURS—EAST SALON

9:45 AM–6:30 PM EXHIBIT HALL OPEN—HALL D

9:45–10:15 AM Exhibit Hall Coffee Social
3:15–3:45 PM Exhibit Hall Coffee Social
5:30–6:30 PM Exhibit Hall Networking Reception
& Poster Session II

ROOM 202
Cancer Gene Therapy
Chair: Christopher LaRocca MD

8:00–8:24 AM CRISPR/Cas-based Editing Approaches to Produce

T-cells Resistant to Immune Suppression
EDUCATION SESSIONS

Theresa Kaeuferle, PhD, LMU Klinikum

8:24–8:48 AM Oncolytic Virotherapy: Progress and Challenges of

Clinical Translation
Julia Davydova, MD, PhD, University of Minnesota

8:48–9:12 AM Updates on NK Cells for Cancer Therapy

Nathan Schloemer, MD, Children’s Wisconsin/Medical
College of Wisconsin

9:12–9:36 AM Optimizing CAR-T Therapy for Brain Tumors

Ryuma Tanaka, MD, Children’s Wisconsin/Medical College
of Wisconsin

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BALLROOM C
CRISPR/Cas9 Gene Editing—Concepts to In Vivo Editing
Chair: Nuria Morral, PhD, Indiana University School of Medicine

8:00–8:24 AM TK
TK

8:24–8:48 AM CRISPR/Cas9-guided Orthotopic T-cell Receptor

Replacement to Engineer Close to Physiological
Antigen-specific T-cells
Dirk Busch, MD,Institute for Medical Microbiology,
Immunology, and Hygiene, Technical University of Munich
EDUCATION SESSIONS

8:48–9:12 AM In Vivo CRISPR Base Editing of PCSK9 Durably Lowers

Cholesterol in Primates
Sekar Kathiresan, MD, Verve Therapeutics

9:12–9:36 AM Base Editing and Prime Editing in Mouse Models

Wen Xue, PhD, University of Massachusetts Medical School

ROOM 207
Integrating Retroviral Vectors
Chair: John Tisdale, MD, National Institutes of Health; National Heart, Lung,
and Blood Institute

8:00–8:32 AM Gene Modified HSCs Bring Despair and Hope

Marina Cavazzana, MD, PhD, Hôpital Necker-Enfants Malades

8:32–9:04 AM Are Globin Vectors Lacking an Insulator Safe?

Michel Sadelain, PhD, Memorial Sloan Kettering Cancer Center

9:04–9:36 AM Potential and Identified Safety Issues With

Integrating Vectors
Melissa Bonner, PhD, bluebird bio

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ROOM 102
Gene- and Cell-based Therapies for Lung and GI
Co-chairs: Maria Limberis, PhD, Spirovant Sciences Inc
and Alisha Gruntman, DVM, PhD, University of Massachusetts Chan Medical School

8:00–8:25 AM Advances in Macrophage-based Cell Therapies: Alveolar

SCIENTIFIC SYMPOSIA

Proteinosis as a Paradigm Disease

Bruce Trapnell, MD, Cincinnati Children’s Hospital
Medical Center

8:25–8:50 AM Cell- and EV-based Approaches for COVID-19 Respiratory

Failure: A Success?
Maroun Khoury, PhD, Center of Interventional Medicine for
Precision and Advanced Cellular Therapy

8:50–9:15 AM Development of Selective Organ Targeting (SORT) Lipid

Nanoparticles (LNPs) for Lung-specific Delivery
Daniel J. Siegwart, PhD, UT Southwestern Medical Center

9:15–9:40 AM Human Mini Lungs Grown in Lab Dishes, Also in GI

Soumita Das, PhD, UC San Diego

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BALLROOM B
Intended and Unintended Roles of Viral Vector Heterogeneity
in Gene Therapy
Co-chairs: Chen Ling, PhD, Fudan University
and Brian Bigger, PhD, University of Manchester

8:00–8:25 AM RNA Virus Genome Diversity: Mechanisms and

Consequences for Therapeutic Vectors
SCIENTIFIC SYMPOSIA

Roberto Cattaneo, PhD, Mayo Clinic

8:25–8:50 AM DNA Virus Heterogeneity—AAV Vectors With Expansion to

Other DNA Viral Vectors
Phillip W.L. Tai, PhD, University of Massachusetts Chan
Medical School

8:50–9:15 AM Lentiviral Vector Heterogeneity in Clinical Applications

Megan D. Hoban, PhD, bluebird bio

9:15–9:40 AM Sizing and Counting Empty and Filled AAV Particles by

High Resolution Native Mass Spectrometry and Single
Molecule Charge Detection Mass Spectrometry
Albert J.R. Heck, PhD, Utrecht University

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SALON G
Musculoskeletal Gene Therapy: Progresses and Challenges
Co-chairs: Scott Q. Harper, PhD, Nationwide Childen’s Hospital
and Chunping Qiao, PhD, REGENXBIO

8:00–8:25 AM Genome Editing and Immuno-engineering

for Arthritis Therapy
Farshid Guilak, PhD, Washington University

8:25–8:50 AM Immunomodulation as an Adjunctive Therapy to AAV

Systemic Dosing to Improve Safety, Increase Expression
SCIENTIFIC SYMPOSIA

and Allow for Repeated AAV Dosing

Manuela Corti, PhD, University of Florida

8:50–9:15 AM Genotype-phenotype Correlations in DMD Patients and

AAV-U7-SnRNA-mediated Exon Skipping for DMD
Kevin M. Flanigan, MD, Nationwide Children’s Hospital

9:15–9:40 AM Directed Evolution of a Family of AAV Capsid Variants

Enabling Potent Muscle-directed Gene Delivery
Across Species
Sharif Tabebordbar, PhD, Kate Therapeutics

BALLROOM A
Fireside Chat | State of the Field: Emerging Regulatory Trends
Chair: Keith Wonnacott, PhD, Lexeo Therapeutics

8:00–9:45 AM Peter Marks, MD, PhD, Food and Drug Administration

Ana Hidalgo-Simon, MD, PhD, European Medicines Agency

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ROOM 204
Symposium on Translating Tissue Engineering and Regenerative
Medicine Approaches into Therapies
Co-chairs: Steven Becker, PhD, National Institutes of Health
and Lloyd F. Rose, PhD, U.S. Army Medical Materiel Development Activity/BioFabUSA
SCIENTIFIC SYMPOSIA

8:00–8:20 AM Decellularized Heart Scaffold

Doris A. Taylor, PhD, RegenMedix Consulting

8:20–8:40 AM Projects Done With the BioFabUSA Tissue Foundry

Tom Bollenbach, PhD, Advanced Regenerative
Manufacturing Institute/BioFabUSA

8:40–9:00 AM Preclinical Spinal Cord Repair Research With

Neural Stem Cells
Mark Tuszynski, MD, PhD, UC San Diego

9:00–9:45 AM Round Table Discussion

Moderator: Laura Ricles, PhD, Food and Drug Administration

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ROOM 206
The Ethical Gray Zone? Perspectives on the Development and Governance
of Gene and Cell Therapies for Human Enhancement
Co-chairs: Eric Juengst, PhD, UNC Chapel Hill
and Benjamin Hurlbut, PhD, Arizona State University

8:00–8:20 AM Introduction: Defining and Differentiating Disease

Versus Enhancement
Sheila Jasanoff, PhD, JD, Harvard University
SCIENTIFIC SYMPOSIA

8:20–8:40 AM The Case for Rethinking Health-related

Enhancements in Specific Social Contexts:
NASA’s Space Radiation Program
Lisa Scott-Carnell, PhD, National Aeronautics and
Space Administration

8:40–9:00 AM Special Ethical Considerations Relevant to Governance

of Enhancement Interventions
Peter Mills, PhD, Nuffield Council on Bioethics

9:00–9:20 AM Reflections on the Treatment Versus Enhancement Debate

Through the Years: Key Points and Lessons Learned for
the Present
John Evans, PhD, UC San Diego

9:20–9:45 AM Panel Discussion

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ROOM 201
Therapeutic Applications of RNA Therapy Strategies
Co-chairs: Lauren E. Woodard, PhD, Vanderbilt University Medical Center
and Loree Heller, PhD, University of South Florida

8:00–8:25 AM In Vivo Cellular Reprogramming by Targeted

SCIENTIFIC SYMPOSIA

mRNA-LNP
Hamideh Parhiz, PharmD, PhD, University of Pennsylvania

8:25–8:50 AM Strategies for Developing mRNA-based Therapeutics

for Rare Diseases
Lisa Rice, PhD, Moderna Therapeutics

8:50–9:15 AM Exosomes in Nucleic Acid Delivery

Susmita Sahoo, PhD, Icahn School of Medicine

9:15–9:40 AM Ultrasound-mediated Gene Delivery (UMGD)

Costas Arvanitis, PhD, Georgia Institute of Technology

SALON H
Late Breaking Oral Abstract Sessions
8:15 AM–9:45 PM

Due to the late-breaking nature of this session, individual presentations were

not yet selected at time of publish.

See [Link]/program for more information

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ROOM 209
Industry Interactions
Bio-Rad Laboratories | Droplet Digital PCR for Advancing Quality
Manufacturing of Gene and Cell Therapies
EXHIBITOR SHOWCASES

8:15–8:45 AM Presenter(s) to be Announced

ROOM 209
Industry Interactions
Catalent Cell & Gene Therapy | AAV Platform Process Accelerating
Production from Gene to Clinic
9:15–9:45 AM George Buchman, PhD
VP, Pre-clinical and Process Development,
Catalent Cell & Gene Therapy

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TUESDAY, MAY 17, 2022
SPONSORED BY:

HALL E
George Stamatoyannopoulos Memorial Lecture and Award Presentation

10:15–10:35 AM ASGCT Update from President Beverly Davidson, PhD

and CEO David Barrett, JD

10:35–10:55 AM Presentation of the Career Development Awards and

KEYNOTE SESSIONS

Diversity & Inclusion Awards

10:55–11:05 AM Presentation of the Sonia Skarlatos Public Service Award

to P.J. Brooks, PhD

11:05–11:15 AM Presentation of the Jerry Mendell Award for Translational

Science to Katherine High, MD

11:15 AM–12:00 PM Keynote Address from Drew Weissman, PhD

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BALLROOM C
Corning Life Sciences | Leveraging a Novel Adherent Cell Culture
Technology for Highly Efficient and Intensified Cell and Gene
Therapy Manufacturing
12:00–1:30 PM Zara Melkoumian, PhD
Business Technology Director, Corning Life Sciences
Hannah Rasby
Sr. Process Development Engineer, Viacyte

Todd Upton, PhD

Innovation Portfolio Sr. Manager, Corning Life Sciences
INDUSTRY SPONSORED SYMPOSIA

BALLROOM B
Cytiva | Cells to Purified Capsids: How to Develop a Scalable rAAV Process

12:00–1:30 PM Åsa Hagner McWhirter

Principal Scientist, R&D, Cytiva

ROOM 207
Taysha Gene Therapies | Accepting the Challenge: Innovative Approaches
and Translational Strategies in Gene Therapy Development

12:00–1:30 PM Suyash Prasad, MBBS, MRCP, MRCPCH, FFPM

Chief Medical Officer, Head of Research and Development,
Taysha Gene Therapies

Steven Gray, PhD

Chief Scientific Advisor and Associate Professor, UTSW Gene
Therapy Program, University of Texas Southwestern

Kimberly Goodspeed, MD
Assistant Professor, Department of Pediatrics,
University of Texas Southwestern

Jagdeep S. Walia, MBBS, FRCPC, FCCMG

Full Professor, Director of Research (Pediatrics),
Queen’s University, Canada

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ROOM 202
INDUSTRY SPONSORED SYMPOSIA

Vertex Pharmaceuticals, Inc. | Breaking Barriers: Managing the Immune

Response to Cell and Gene Therapies

12:00–1:30 PM John F. DiPersio, MD, PhD

Director, Center for Gene and Cellular Immunotherapy,
Washington University School of Medicine
Leslie S. Kean, MD, PhD
Director, Pediatric Stem Cell Transplant Program,
Dana-Farber Cancer Institute
Barry J. Byrne, MD, PhD
Director, Powell Gene Therapy Center, University of Florida
Mike Cooke
Senior Vice President, Cell and Genetic Therapy Research
Team, Vertex Pharmaceuticals Inc.

ROOM 209
Industry Interactions
Bio-Techne | Characterization of Gene Therapy Biodistribution
and Function - From AAV to ASO
eEXHIBITOR SHOWCASES

12:00–12:30 PM Michaeline Bunting, PhD

Director, Spatial Biology Division, Bio-Techne

ROOM 209
Industry Interactions
Precision for Medicine | Key Considerations for Design and
Implementation of NAb Bioassays in Gene Therapy Development

1:00–1:30 PM Travis Harrison, PhD

Vice President, Bioassay Solutions, Precision for Medicine

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TUESDAY, MAY 17, 2022
SPONSORED BY:

HALL E | 1:30–3:00 PM
Outstanding New Investigator Symposium

SOUTH PRE-FUNCTION
Networking Event—Career Fair
3:00–5:00 PM

Connect with employers and get a complimentary professional headshot!

Make sure you are registered for the Annual Meeting to attend the Career Fair.

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ROOM 209
Industry Interactions
Co-chairs: Blythe Sather, PhD and Le Cong, PhD, Stanford University

3:30–3:45 PM PROGEN | AAV Lateral Flow Test for Rapid Titer

Determination
Dana Holzinger, Head of Product Management, PROGEN

3:45–4:00 PM Refeyn | SamuxMP - A Mass Photometer for Rapid

AAV Analytics
Gareth Rogers, Product Manager, Refeyn
TOOLS AND TECHNOLOGY FORUM II

4:00–4:15 PM MYRIADE | VIDEODROP: Ideal Tool for Lentiviral Vector

Bioproduction Follow-up
Marie Berger, PharmD, Application Engineer, MYRIADE

4:15–4:30 PM SIRION Biotech | Taking Gene Delivery to the

Single Cell Level
Christian Thirion, PhD, CEO and Founder, SIRION Biotech

4:30–4:45 PM Curiox Biosystems

4:45–5:00 PM Wyatt Technology | Biophysical Characterization

and Quality Control of Vaccines and Gene Vectors With
Light Scattering
William Penny, PhD, Regional Account Manager &
Application Scientist, Wyatt Technology

5:00–5:15 PM Mission Bio | Harnessing Single-cell Multi-omics to

Advance Cell and Gene Therapy Research
Yue Wang, PhD, Business Development Manager, Cell and
Gene Therapy, Mission Bio

5:15–5:30 PM Synthego | Industrialized CRISPR: Accelerating

Disease Research
Peter Deng, PhD, Associate Product Manager, On-Market
Strategy, Synthego

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TUESDAY, MAY 17, 2022

5:30–5:45 PM PackGene Biotech INC | π-AlphaTM 293 AAV High-yield

TOOLS AND TECHNOLOGY FORUM II

Production Technology to Improve Scalability

and Biosafety
Baker Lu, PhD, Business Development Director,
North America, PackGene Biotech INC

5:45–6:00 PM Unchained Labs | Hit the Gas Pedal on AAV Analysis and
Prep with Uncle, Stunner and Big Tuna
Kevin Lance, PhD, Director of Analytics Marketing,
Unchained Labs

CELL & GENE THERAPY

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A partnership, every step of the way, for viral vector manufacturing of your gene
therapy products. From process development up to commercial launch,Yposkesi is the
full-service CDMO of choice, working by your side, to meet your timelines and budget.

Contact us to discover how we can help you to reach your goals.

[Link]
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BALLROOM A
Discoveries in Fundamental AAV Biology
Co-chairs: Nicole Paulk, PhD, UCSF and Anna Maurer, PhD, UC Berkeley

3:45–4:00 PM 433: Structural Characterization of Patient-derived

Anti-AAV9 Monoclonal Antibodies Generated
Post-Zolgensma Treatment
Mario Mietzsch, PhD, University of Florida

4:00–4:15 PM 434: Characterization of Alternative Reading Frame

Proteins Generated from AAV Cassettes
ORAL ABSTRACT SESSIONS

Ferzin Sethna, Ph.D, Asklepios Biopharmaceuticals

4:15–4:30 PM 435: Transcription, Translation, and Immunogenic Potential

of P5-Associated rAAV Contaminants Post-infection
Mark Brimble, PhD, St Jude Children’s Research Hospital

4:30–4:45 PM 436: Unlocking Avian AAV Transduction in Mammalian

Cells and Tissues for Immune Evasion and Redosing
Ezra Loeb, Duke University

4:45–5:00 PM 437: Genome Packaging Efficiencies of Anc80 and AAV9

Vectors using Non-canonical Rep-ITR Combinations
Anusha Sairavi, Oregon Health and Science University

5:00–5:15 PM 438: AAV Capsid Dynamics at the 5-fold Pore Controls

Genome Release
Joshua Hull, BS, University of Florida

5:15–5:30 PM 439: Furin is a Host Factor Restricting Adeno-associated

Virus 4 Transduction
Timothy Smith, Duke University

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SALON G
Enhanced AAV Targeting
Chair: Ana Rita Batista, PhD, UMass Chan Medical School

3:45–4:00 PM 440: Effects of an Early Gene Therapy Targeting

Oligodendrocytes in a Mouse Model of
Adrenomyeloneuropathy
Yasemin Gunes, UMR 1195 Inserm and University
Paris Saclay

4:00–4:15 PM 441: Engineered AAV Capsids Exhibit Improved

Transduction of the Central Nervous System after
CSF Administration in Adult Cynomolgus Macaques
David Ojala, Sangamo Therapeutics, Inc.
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 442: A Direct Comparison of Five AAV Capsids for

Intramuscular Inoculation in Non-human Primates
Matthew Gardner, PhD, Emory University

4:30–4:45 PM 443: Systemic Administration of Novel Engineered AAV

Capsids Facilitates Enhanced Transgene Expression in the
Macaque Central Nervous System
Alexandra Stanton, Broad Institute

4:45–5:00 PM 444: Astrocyte-restricted Gene Silencing Improves

the Safety of AAV9-Mediated Gene Therapy for
Alexander Disease
Wassamon Boonying, PhD, University of Massachusetts
Chan Medical School

5:00–5:15 PM 445: Positron Emission Tomography I-124-labeled AAV

Assessment of CSF to Blood Diffusion and Consequent
Systemic Distribution of AAV Capsids Following CSF
Administration of AAV Vectors
Jonathan Rosenberg, MD, Weill Cornell Medicine

5:15–5:30 PM 446: Identification of New AAV9 Engineered Capsids

Targeting Mouse and Monkey Brains Through a Directed
Evolution Approach in BALB/c Mice
Giannelli Serena, PhD, Fondazione Centro San Raffaele

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HALL E
Gene Editing in Cancer and Complex Diseases
Co-chairs: Angelo Lombardo, PhD, SR-TIGET
and Mara Pavel-Dinu, PhD, Stanford University

3:45–4:00 PM 447: Targeting the Hepatitis BcccDNA with a Sequence-

Specific ARCUS Nuclease to Eliminate Hepatitis B Virus
In Vivo
Cassandra Gorsuch, PhD, Precision BioSciences

4:00–4:15 PM 448: A Novel DNA Oligo-based Repair Strategy for the

Functional Correction of Shwachman-Diamond Syndrome
ORAL ABSTRACT SESSIONS

CY Zhang, PhD, Dana-Farber Cancer Institute

4:15–4:30 PM 449: Liver Gene-editing Based on Nickase Cas9 for the

Treatment of Primary Hyperoxaluria Type I (PH1) is More
Efficient when Using an All-in-one Delivery System
Laura Torella, Center for Applied Medical Research (CIMA),
University of Navarra

4:30–4:45 PM 450: Enhanced CRISPR/Cas9 Genome Editing in Heart and

Skeletal Muscle with a Potent New AAV Variant
Trevor Gonzalez, Duke University

4:45–5:00 PM 451: A MiniCEP290 Gene Replacement Therapy to Treat

CEP 290-Leber Congenital Amourosis (LCA10)
Bhubanananda Sahu, PhD, Iveric Bio

5:00–5:15 PM 452: A Novel Polyfunctional Editing Strategy for Adoptive

T-cell Immunotherapy of Cancer
Tania Baccega, IRCCS San Raffaele Scientific Institute

5:15–5:30 PM 453: Generation of Efficient Lipid Nanoparticles for

Liver-Directed Gene Therapy and Genome Editing
Claude Warzecha, PhD, University of Pennsylvania

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SALON H
Physical Methods and Extracellular Vesicle-Based Gene Transfer
Chair: Richard Heller, PhD, University of South Florida

3:45–4:00 PM 454: DNA Sensor Palmitoylation in Mouse Skeletal Muscle

Following DNA Electroporation
Amanda Sales Conniff, PhD, University of South Florida

4:00–4:15 PM 455: Novel Non-thermal Plasma Based Delivery of

Plasmid DNA
Pavan Cherukuri, PhD, University of South Florida
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 456: Electrotransfer Combined with Moderate Heat and

Impedance Monitoring to Enhance Delivery of Agents to
Multiple Tissues
Richard Heller, PhD, University of South Florida

4:30–4:45 PM 457: Ultrasound Mediated Gene Delivery Specifically

Targets Liver Sinusoidal Endothelial Cells for Sustained
FVIII Expression in Hemophilia A Mice
Savannah Lawton, Seattle Children’s Research Institute

4:45–5:00 PM 458: Assessment of Commensal E. coli Outer Membrane

Vesicles for Application in a Novel Oral Delivery System
Kari Heck, University of Nebraska-Lincoln

5:00–5:15 PM 459: Extracellular Vesicle-mediated Therapeutic

Delivery of RIG-I Agonists for Immunotherapy Against
Breast Cancer
Minh T.N. Le, PhD, Department of Pharmacology, Yong Loo
Lin School of Medicine, National University of Singapore

5:15–5:30 PM 460: Engineering Cells to Produce miRNA-loaded

Exosomes for Potential Biotherapeutics
Andrew Hamann, PhD, University of Nebraska-Lincoln

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ROOM 201
Inborn Errors of Metabolism Gene and Cell Therapies:
Proof-of-Concepts and Beyond
Co-chairs: Gerald Lipshutz, MD, David Geffen School of Medicine at UCLA
and Stephanie Cherqui, PhD, UC San Diego

3:45–4:00 PM 461: Safety and Efficacy of a Dual-function AAV9

BCKDHA-BCKDHB Gene Replacement Vector in Murine
and Bovine Models of Classic Maple Syrup Urine Disease
Kevin Strauss, MD, Clinic for Special Children

4:00–4:15 PM 462: Homology Independent Targeted Integration

Leads to Highly Efficient Protein Expression and Secretion
From Liver
Federica Esposito, TIGEM
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 463: Safety and Efficacy of DTX301 in Adults With

Late-Onset Ornithine Transcarbamylase (OTC) Deficiency:
A Phase 1/2 Trial
Cary Harding, MD, Oregon Health & Science University

4:30–4:45 PM 464: Genome Editing in a Canine Model for Glycogen

Storage Disease Type Ia
Benjamin Arnson, Duke University School of Medicine

4:45–5:00 PM 465: Liver Directed Lentiviral Gene Therapy Ameliorates

the Phenotype of Progressive Familiar Intrahepatic
Cholestasis Type 2 in a Mouse Model
Elena Barbon, PhD, San Raffaele Telethon Institute for Gene
Therapy, IRCCS San Raffaele Scientific Institute

5:00–5:15 PM 466: Nuclease Enhancement of AAV Mediated Editing into

Albumin in Neonatal Mice with Methylmalonic Acidemia
(MMA)
Leah Venturoni, PhD, NHGRI

5:15–5:30 PM 467: Updated Interim Results of Transpher A,

a Multicenter, Single-Dose, Pivotal Clinical Trial of
ABO-102 Gene Therapy for Sanfilippo Syndrome Type A
(Mucopolysaccharidosis IIIA)
Kevin M. Flanigan, MD, Nationwide Children’s Hospital

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ROOM 204
Applications of Improved Gene Therapy Methods in Neurologic Disorders
Co-chairs: Kathrin Meyer, PhD, Nationwide Children’s Hospital
and Steven Gray, PhD, UT Southwestern

3:45–4:00 PM 468: Intrathalamic Delivery of [Link] Rescues

Pathology in GRN Null Mice and Achieves Widespread
Cortical Expression in a Large Animal Model Without
Expression in the Liver
Christopher Shaw, MD, Kings College London

4:00–4:15 PM 469: AAV-based GDNF Expression in VTA Prevents Relapse

to Alcohol-Drinking Behavior and Modifies Mesolimbic
ORAL ABSTRACT SESSIONS

Dopamine Function in Rhesus Macaques: A Gene Therapy

Approach to Treating Alcohol Use Disorder
Victor Van Laar, PhD, The Ohio State University

4:15–4:30 PM 470: Vectorized Delivery of Tau Reduction Therapy as

a Treatment Approach for Tauopathies
Rachel Bailey, PhD, UT Southwestern Medical Center

4:30–4:45 PM 471: CAP-001: Systemic AAV Gene Therapy With Next

Generation Capsids for MPS II Disease
Nicholas Flytzanis, PhD, Capsida Biotherapeutics, Inc.

4:45–5:00 PM 472: Durable and Specific Rescue of UBE3A Expression in

the Brain of an Angelman Syndrome Mouse Model Using
an Artificial Transcription Factor
Henriette O’Geen, PhD, UC Davis

5:00–5:15 PM 473: Gene Therapy Mediated Cross Correction for CDKL5

Deficiency Disorder
Heather Born, PhD, University of Pennsylvania

5:15–5:30 PM 474: Safe And Efficacious Rescue Of GM3 Synthase

Deficiency Mice By Spatially Regulated Gene Delivery
Huiya Yang, UMass Chan Medical School

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ROOM 102
Enhancing CAR T-cell Efficacy
Co-chairs: Craig Sauter, MD, MSKCC
and Barbara Savoldo, MD, PhD, UNC Lineberger Comprehensive Cancer Center

3:45–4:00 PM 475: Leucine Zipper-based Cytokine Receptors Augments

CAR T-cell Immunotherapy for Solid Tumors
Matthew Bell, St. Jude Children’s Research Hospital

4:00–4:15 PM 476: Epigenome Editing Enables PD1 Silencing in

CAR T-cells
Maria Silvia Roman Azcona, PhD student, Institute for
ORAL ABSTRACT SESSIONS

Transfusion Medicine and Gene Therapy

4:15–4:30 PM 477: TNFR2 as a Target to Improve CD19-Directed

CAR T-cell Fitness and Antitumor Activity in
Large B-cell Lymphoma
Claudia Manriquez Roman, [Link]., Mayo Clinic

4:30–4:45 PM 478: A High Expression of IL15 Receptor Alpha (IL15Rα) in

Glioblastoma Microenvironment Enables IL15-armored
CAR T-cells to Modulate Tumor Immunosuppression and
Improve Survival in Syngeneic Models
Irina V. Balyasnikova, PhD, Northwestern University Feinberg
School of Medicine

4:45–5:00 PM 480: Allogeneic Donor-derived CD19-Chimeric Antigen

Receptor (CAR) T-cells for Relapsed B-cell Malignancies
After Hematopoietic Stem Cell Transplantation
Ibrahim Muhsen, MD, Houston Methodist Hospital

5:00–5:15 PM 481: High-Affinity PD1-CD28 Chimeric Switch Receptors

Enhance Costimulatory Signaling and Improve TCR and
CAR T-cell Antitumor Activity
Brooke Prinzing, PhD, St. Jude Children’s Research Hospital

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ROOM 202
Gene Therapy for Immunologic Diseases
Co-chairs: Donald Kohn, MD, UCLA
and Claire Booth, MBBS, PhD, UCL Great Ormond Street Institute of Child Health

3:45–4:00 PM 482: A Novel Engineered CRISPR-Associated Nuclease

Accurately Removes ELANE Mutated Allele and Shifts
HSC Differentiation Towards Neutrophils in Severe
Congenital Neutropenia
Rafi Emmanuel, PhD, Emendo Biotherapeutics

4:00–4:15 PM 483: Editing T-cell Repertoire by Thymic Epithelial

Cell-Directed Gene Transfer Abrogates Risk of Type 1
Diabetes Development
Andrea Annoni, PhD, San Raffaele Telethon Institute for
ORAL ABSTRACT SESSIONS

Gene Therapy

4:15–4:30 PM 484: Therapeutic Gene Editing of T-cells Corrects

CTLA4 Insufficiency
Thomas Fox, MD, UCL

4:30–4:45 PM 485: A Simultaneous Knock-out Knock-in Gene Editing

Strategy in HSPCs Potently Inhibits R5- and X4-tropic
HIV Replication
Amanda Dudek, PhD, Stanford University School of Medicine

4:45–5:00 PM 486: Transcriptional Mapping of Human Hematopoietic

Stem and Progenitor Cells discriminates Chronic
Granulomatous Disease Patients Able to Benefit From
Gene Therapy Treatment
Steicy Sobrino, Imagine Institute

5:00–5:15 PM 487: Base Editing of Hematopoietic Stem Cells Rescues

T-cell Development for CD3d Severe Combined
Immunodeficiency
Grace McAuley, UCLA

5:15–5:30 PM 488: Chemotherapy-free Engraftment of Gene Edited

Human Hematopoietic Stem Cells Leveraged on
Mobilization and mRNA-based Engineering
Attya Omer Javed, IRCCS San Raffaele Scientific Institute

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ROOM 206
Immune Responses to Gene Delivery and Vaccine Approaches
Co-chairs: Matthew Gardner, PhD, Emory University
and Manish Muhuri, PhD, Biogen

3:45–4:00 PM 489: Loss of AAV-FVIII Gene Therapy in Hemophilia A Mice

Due to Shutdown of Hepatic Protein Production Rather
Than Loss of Vector or Transduced Cells
Kentaro Yamada, PhD, Indiana University School of Medicine

4:00–4:15 PM 490: Novel Early Checkpoint Modifier Demonstrates

Broadened and Enhanced CD8+ T-cell Responses Across
Multiple Preclinical Studies
Hildegund Ertl, The Wistar Institute
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 491: Mucosal Chemokine CCL27 Adjuvant Uniquely

Improves Mucosal Responses to SARS-CoV-2 synDNA
Antigens Providing Heterologous Protection Against Delta
Variant Challenge
Ebony Gary, PhD, The Wistar Institute

4:30–4:45 PM 492: An Intranasal saRNA/NLC Vaccine Induces Robust

Mucosal and Systemic Immunity to SARS-CoV-2 in Mice
Emily Voigt, PhD, Infectious Disease Research Institute

4:45–5:00 PM 493: AAV-mediated Expression of Monoclonal Antibodies

Provides Protection in a Mouse Model of Marburg Virus
Infection and Long-term Expression in an Ovine Model
Amira Rghei, BS, University of Guelph

5:00–5:15 PM 494: Adenovirus Capsid Proteins-based Anti-fentanyl

Vaccine Attenuates Fentanyl-induced Behaviors in Mice
Bishnu De, Weill Cornell Medicine

5:15–5:30 PM 495: Pre-existing Maternal Humoral Immunity to

Adeno-Associated Virus Impairs Fetal Gene Editing in a
Serotype-specific Fashion
John Riley, Children’s Hospital of Philadelphia

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BALLROOM C
Anemocyte | Expert Talk—My Letter to Santa: Wishes and Reality
Impacting Cell and Gene Therapies

5:30–7:00 PM Federico Mingozzi, PhD, MBA

Chief Scientific Officer; Spark Therapeutics

Julianne Smith, PhD

Chief Development Officer, GeneSpire

Nathalie Belmonte, PhD

SVP Research & Translation, Quell Therapeutics

Jim Faulkner, PhD

INDUSTRY SPONSORED SYMPOSIA

Chief Technical Officer, Ascidian Therapeutics

Miguel Forte, MD, PhD

CEO, Bone Therapeutics, President-Elect ISCT, International
Society for Cell & Gene Therapy

Luigi Naldini, MD, PhD

Co-founder, Genenta Science; Director of SR-Tiget,
San Raffaele Telethon Institute for Gene Therapy

ROOM 207
Charles River Laboratories | Thinking With the End in Mind:
How to Accelerate C&GT Research and Development

5:30–7:00 PM Sam Chuang, PhD

Director, Scientific Advisory Services,
Charles River Laboratories
Matt Hewitt
Executive Director, Scientific Services Cell & Gene Therapy,
Charles River Laboratories
Steven Miklasz
Strategic Key Account & Technical Development Director,
Charles River Laboratories
Dimpi Patel
Senior Director Global Cell & Gene Therapy Compliance,
Charles River Laboratories

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BALLROOM B
QIAGEN LLC | Industry Sponsored Symposium

5:30–7:00 PM Miriam Menezes

Senior Scientist - Molecular Biology/Automation,
Spark Therapeutics
INDUSTRY SPONSORED SYMPOSIA

John Kerwin
Technical Head, Gene Therapy Franchise,
National Resilience

Dana Cipriano
VP of Testing and Analytical Services,
The Center for Breakthrough Medicines

Mandy Conver
Associate Director, dPCR Commercial Operartions,
QIAGEN LLC

A’Drian Pineda
Sr. Business Development Manager, Biopharma,
QIAGEN LLC

Victoria Best
Director, Analytical Sciences, AmplifyBio

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HALL D
Networking Reception & Poster Session II
5:30–6:30 PM

ROOM 103
Networking Event—New Member Meetup
6:00–8:00 PM

The New Member Mixer welcomes attendees who have joined ASGCT over the
past three years. Usually reserved for members joining in the past 12 months,
we’ve expanded the mixer to include those who have joined since we were
last in-person in 2019. This informal setting will introduce new members to
the Society and highlight the many ways they can use our resources as their
professional hub. Attendees will also hear from a longtime member who has
adeptly used their Society membership throughout their career.

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WEDNESDAY, MAY 18, 2022

7:00 AM–6:00 PM REGISTRATION HOURS—EAST SALON

9:45 AM–6:30 PM EXHIBIT HALL OPEN—HALL D

9:45–10:15 AM Exhibit Hall Coffee Social
3:15–3:45 PM Exhibit Hall Coffee Social
5:30–6:30 PM Exhibit Hall Networking Reception
& Poster Session III

BALLROOM B
Gene Therapy Beyond Cancer
Chair: Satiro Nakamura de Oliveira, MD, UCLA

8:00–8:24 AM Gene Therapies for Inherited Bleeding Disorders

EDUCATION SESSIONS

David A. Wilcox, PhD, Medical College of Wisconsin;

Children’s Research Institute-Children’s Wisconsin;
Versiti Blood Research Institute

8:24–8:48 AM Gene Therapies in Hemoglobinopathies

Daniel Bauer, MD, PhD, Boston Children’s Hospital

8:48–9:12 AM Gene Therapies for Liver Disease

Gloria Gonzalez-Aseguinolaza, PhD, CIMA-University of
Navarra, Vivet Therapeutics

9:12–9:36 AM Genetically Engineered CAAR T-cell Therapies

for B Cell-mediated Autoimmune Diseases
Aimee S. Payne, MD, PhD, University of Pennsylvania

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ROOM 204
Pre-clinical Models
Co-Chair: Erik Barton, PhD, Pfizer,
and Hildegard Buning, PhD, Hannover Medical School
EDUCATION SESSIONS

8:00–8:24 AM Small Animal Models for Gene Therapy Research

Karl-Dimiter Bissig, MD, PhD, Duke University

8:24–8:48 AM Immuno-oncology Humanized Mice and PDX

Saar Gill, MD, PhD, University of Pennsylvania

8:48–9:12 AM Immune Competent Pet Dogs with Spontaneous Disease

as Pre-clinical Models for Cellular Therapy
Nicola Mason, BVetMed, PhD, University of Pennsylvania

9:12–9:36 AM FDA/CBER Perspective for In Vivo Preclinical Testing

of Cell and Gene Therapy Products
Abigail L. Shearin, VMD, PhD, OTAT, Food and
Drug Administration

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WEDNESDAY, MAY 18, 2022

ROOM 206
Combating Misinformation in Science
Co-chairs: Karen Bulaklak, PhD, Sarepta Therapeutics
and Edith Pfister, PhD, UMASS Chan Medical School
SCIENTIFIC SYMPOSIUM

8:00–8:24 AM Misinformation in and About Science

Jevin West, PhD, University of Washington Center f
or an Informed Public

8:24–8:48 AM California’s Initiatives to Advance Precision Medicine

Julianne McCall, PhD, California Governor’s Office of
Planning and Research

8:48–9:12 AM Breaking Down Gene Therapy and CRISPR Topics

for Non-scientific Audiences
Jennifer E. Adair, PhD, Fred Hutchinson Cancer
Research Center

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ROOM 102
Challenges and Status in Development of Gene Therapies in Low- and
Middle-income Countries (LMICs)
Co-chairs: Richard Koya, MD, PhD, University of Chicago School of Medicine
and Savita Rangarajan, MD, University Hospital Southampton

8:00–8:20 AM Investigator Perspective on Improving Transportability

Jennifer E. Adair, PhD, Fred Hutchinson Cancer
SCIENTIFIC SYMPOSIUM

Research Center

8:20–8:40 AM Bringing Safe, Effective, and Accessible Gene Therapies for

HIV and Sickle Cell Disease to LMICs
Mike McCune, MD, PhD, UCSF

8:40–9:00 AM Challenges of Initiating Trials in LMICs

Cissy Kityo, MD, Joint Clinical Research Center, Uganda

9:00–9:20 AM Manufacturing: Barriers and Solutions to LMICs

Kollengode V. Subramanian, Reliance Life Sciences, India

9:20–9:45 AM Panel Discussion

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BALLROOM A
Gene Editing: New Technology Advances
Co-chairs: Thomas J. Cradick, PhD, Excision BioTherapeutics
and Paula Rio, PhD, Centro de Investigaciones Energéticas,
Medioambientales y Tecnológicas (CIEMAT)
SCIENTIFIC SYMPOSIUM

8:00–8:25 AM Prime Editing: Precision Gene Editing

Without Double-strand DNA Breaks
Peter Chen, MD, PhD, Harvard University

8:25–8:50 AM Heritable Control of Gene Expression by CRISPR

Epigenome Editing
James K. Nuñez, PhD, UC Berkeley

8:50–9:15 AM Compact CRISPR Proteins for Genome Editing

Pei-Qi Liu, Mammoth Biosciences

9:15–9:40 AM Programmable Gene Insertion (PASTE)

Omar Abudayyeh, PhD, MIT
Jonathan Gootenberg, PhD, MIT

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SALON G
Inborn Metabolic Issues
Co-chairs: Anne Galy, PhD, Genethon
and Alessandro Aiuti, MD, PhD, San Raffaele Telethon Institute

8:00–8:26 AM Clinical Trial Results of Hematopoietic Stem Cell Gene

SCIENTIFIC SYMPOSIUM

Therapy for Mucopolysaccharidosis Type I Hurler

Maria Ester-Bernardo, MD, PhD, San Raffaele
Telethon Institute

8:26–8:52 AM Pre-clinical Studies of Gene Therapy for Inherited

Liver Disorders
Gloria Gonzalez-Aseguinolaza, MD, PhD, CIMA,
University of Navarra

8:52–9:18 AM Clinical Trials of AAV for Aromatic L-amino Acid

Decarboxylase Deficiency (AADC)
Paul Wuh-Liang Hwu, MD, PhD, National Taiwan
University Hospital

9:18–9:44 AM Pre-clinical Development of Lentiviral Liver Gene Therapy

for Pediatric Metabolic Diseases
John Counsell, PhD, University College London

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ROOM 207
Non-viral Delivery: A Diverse Toolbox Comes of Age
Co-chairs: Stefan Radtke, PhD, Fred Hutchinson Cancer Research Center
and Chantal Pichon, PhD, Center for Molecular Biophysics, CNRS

8:00–8:25 AM Engineered Exosomes as a Delivery System From Bench

to Bedside
Sriram Sathy, PhD, Codiak Biosciences Inc.

8:25–8:50 AM LNP Formulation Engineering for Lung and Endothelial

Ross Wilson, PhD, UC Berkeley

8:50–9:15 AM SEND Harnesses Natural Proteins

Feng Zhang, PhD, Massachusetts Institute of Technology
SCIENTIFIC SYMPOSIA

9:15–9:40 AM Gene-activated Scaffolds for Bone Fracture

Fergal J. O’Brien, PhD, Royal College of Surgeons in Ireland

ROOM 201
Overview of Cardiac Gene Therapy
Co-chairs: Jeff L. Ellsworth, PhD, Stellar Research and Development, LLC
and Ryan L. Boudreau, PhD, University of Iowa

8:00–8:25 AM Pre-clinical Development of Gene Therapy for

Post-operative Atrial Fibrillation
J. Kevin Donahue, MD, University of Massachusetts Chan
Medical School

8:25–8:50 AM Gene-edited Human Stem Cell-derived Cardiomyocytes

for Cardiac Regeneration
Silvia Marchiano, PhD, University of Washington

8:50–9:15 AM Non-viral Delivery of Genes Into the Heart

Valeria Chiono, PhD, Politecnico di Torino

9:15–9:40 AM MicroRNA Manipulation to Control

Myocardial Compliance
Ryan L. Boudreau, PhD, University of Iowa

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ROOM 202
Vector-associated Neural and Ocular Inflammation
Co-chairs: Christine N. Kay, MD, Vitreoretinal Associates
and Paul A. Sieving, MD, PhD, UC Davis

8:00–8:25 AM Strategies To Evade Gene Therapy Inflammation

Ying Kai Chan, PhD, Harvard University
SCIENTIFIC SYMPOSIA

8:25–8:50 AM Identifying and Preventing Unexpected Impacts of

AAV-mediated Gene Delivery in the Nervous System
Lisa M. Boulanger, PhD, Princeton University

8:50–9:15 AM AAV-RS1 Trials for X-linked Retinoschisis: Disease

Condition, Trial Parameters, and Immune Status
Paul Sieving, MD, PhD, UC Davis

9:15–9:40 AM Ocular Inflammation in AAV-mediated Clinical Trials

Christine N. Kay, MD, Vitreoretinal Associates

BALLROOM C
Late Breaking Oral Abstract Sessions
8:00 AM–9:45 PM

Due to the late-breaking nature of this session, individual presentations were

not yet selected at time of publish.

See [Link]/program for more information.

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ROOM 209
Industry Interactions
Informa Pharma Intelligence | The State of Advanced Genetic Therapies
in 2022
EXHIBITOR SHOWCASES

8:15–8:45 AM Ly Nguyen-Jatkoe, PhD

Executive Director, Americas, Informa Pharma Intelligence

Daniel Chancellor
Director, Thought Leadership and Consulting;
Informa Pharma Intelligence

ROOM 209
Industry Interactions
Curiox Biosystems | Exhibitor Showcase

9:15–9:45 AM Presenter(s) to be Announced

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HALL E | 10:15–11:15 AM
Outstanding Achievement Award Symposium
Co-chairs: Beverly L. Davidson, PhD, Children’s Hospital of Philadelphia
and Donald B. Kohn, MD, UCLA
PLENARY SESSIONS

HALL E
Founder’s Award Presentaion
Chair: Beverly L. Davidson, PhD, Children’s Hospital of Philadelphia

11:15 AM–12:00 PM Towards Scalable In Vivo Gene Editing

Francis S. Collins, MD, PhD

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ROOM 202
Dyno Therapeutics | Transforming the Gene Therapy Landscape
With AI-powered AAV Vectors

12:00–1:30 PM Nicole Paulk, PhD

INDUSTRY SPONSORED SYMPOSIA

Assistant Professor of AAV Gene Therapy, UCSF

Eric Kelsic, PhD

CEO, Dyno Therapeutics

Jamie Kwasnieski, PhD

Head of Applied Biology, Dyno Therapeutics

Jeff Gerold, PhD

Head of Data Science, Dyno Therapeutics

Sylvain Lapan, PhD

Head of Gene Therapy, Dyno Therapeutics

Yvette Leung, MBA

Head of Corporate Development, Dyno Therapeutics

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ROOM 207
Miltenyi Biotec | State-of-the-art Closed System Manufacturing of Cell
and Gene Therapy Products With the CliniMACS Prodigy®

12:00–1:30 PM Ian Johnston, PhD

INDUSTRY SPONSORED SYMPOSIA

Industrial and Academic Cooperations Manager,

Senior Project Manager, Miltenyi Biotec

Kunal Patel, MS
Manager, Process Engineering, Miltenyi Biotec

BALLROOM B
Thermo Fisher Scientific | One Workflow, Two Approaches

12:00–1:30 PM Julia Braun

Staff Scientist, Thermo Fisher Scientific

Celine Martin
Senior Product Manager, Thermo Fisher Scientific

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ROOM 209
EXHIBITOR SHOWCASES

Industry Interactions
L7 Informatics | Digitalization – Implementing a Unified Platform
to Enable Transformative Progress for Cell and Gene Therapy Timelines

12:00–12:30 PM Matthew Hewitt, BA, PhD

Executive Director, Scientific Services Cell and Gene
Therapy, Charles River
Vasu Rangadass, PhD
President & CEO, L7 Informatics

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SPONSORED BY:

HALL E
Presidential Symposium and Presentation of Top Abstracts
Chair: Beverly L. Davidson, PhD, Children’s Hospital of Philadelphia

1:30–2:15 PM Turning Genes into Medicines: The Long

and Winding Road from Gene Discovery to
Gene Therapeutics
Katherine A. High, MD, Ask Bio

2:15–2:30 PM 808: Short- and Long-Term Hematopoietic

Reconstitution After Transplantation is Stem Cell-
PLENARY SESSIONS

Driven and Stochastic: Implications for Gene Therapy

Stefan Radtke, PhD, Fred Hutchinson Cancer
Research Center

2:30–2:45 PM 809: Correction of the Sickle Cell Mutation by In Vivo

HSC Prime Editing in a Mouse Model
Chang Li, PhD, University of Washington

2:45–3:00 PM 810: Generation of Islet-specific Engineered

Regulatory T-cells (EngTregs) for Immune Tolerance
Induction in Type 1 Diabetes using a Novel Dual-
editing Strategy
Martina Hunt, Seattle Childrens Research Institute

3:00–3:15 PM 811: AAV-Meganuclease-Mediated Gene Targeting

Achieves Efficient and Sustained Transduction in
Newborn and Infant Macaque Liver
Lili Wang, University of Pennsylvania

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ROOM 209
Industry Interactions
Co-chairs: Kah Whye Peng, PhD
and Rachel M. Bailey, PhD, UT Southwestern Medical Center
3:30–3:45 PM 908 Devices | Actionable Information of Your Cell Culture
Media to Boost Viral Vector Production
Milla Neffling, PhD, Bioprocessing market segment manager,
908 Devices
3:45–4:00 PM Univercells Technologies | An Innovative Platform for
Integrated Continuous Viral Vector Production
Tania Pereira Chilima, Chief Technology Officer,
Univercells Technologies
TOOLS AND TECHNOLOGY FORUM III

4:00–4:15 PM VIVEbiotech | VIVEbiotech’s Approach to Lentiviral Vector

Manufacturing
Natalia Elizalde, PhD, Business Development Director, VIVEbiotech
4:15–4:30 PM Forge Biologics | Platform Process Development for
AAV Manufacturing
David Dismuke, PhD, Chief Technical Officer, Forge Biologics

4:30–4:45 PM SCIEX | Characterization of Lentiviral Vectors Using

Capillary Electrophoresis Platform Technology
Yan Lu, Mass Spec Analytic Scientist I, St. Jude Children’s
Research Hospital
4:45–5:00 PM Virica Biotech | Overcoming Barriers in Viral Vector
Manufacturing: Small Molecule Targeting of
Antiviral Defenses
Jean-Simon Diallo, PhD, Scientific Founder/CEO, Virica Biotech
5:00–5:15 PM 10x Genomics | Bridging the Worlds of Histology and
Genomics with Visium CytAssist
Spontaneous Russell, Senior Product Manager,
Visium Spatial Solutions, 10x Genomics
5:15–5:30 PM Precision NanoSystems | Rethink Genome Editing of
CAR T-cells: Insights into Lipid Nanoparticle Delivery
and Scalable Microfluidic Manufacturing
Angela Zhang, PhD, Senior Product Manager, Precision NanoSystems
5:30–5:45 PM NanoView Biosciences | Purification-Free Detection and
Analysis of Viruses with ExoView®
Clayton Deighan, PhD, NanoView Biosciences

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SALON H
RNA Virus Vectors
Co-chairs: John Tisdale, MD, NIH, NHLBI and Mario Amendola, PhD, Genethon

3:45–4:00 PM 812: Development of a Lentiviral Vector Mediated B-cell

Gene Therapy Platform for the Delivery of the Anti-HIV
eCD4-Ig Immunoadhesin
Eirini Vamva, Scripps Research Institute

4:00–4:15 PM 813: Taking a Good Look Under the Hood of Engineered

Lentiviruses: Nanoview’s Novel LentiView Technology
Allows the Quantitative Profiling of Pseudotyped
Lentiviral Particles
George Daaboul, PhD, NanoView Biosciences Ltd
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 814: Investigating Liver Tissue Dynamics to Improve

In Vivo Gene Therapy with Lentiviral Vectors
Francesco Starinieri , PhD, SR-TIGET

4:30–4:45 PM 815: CD90-Targeted Viral Vectors of Hematopoietic Stem

Cell Gene Therapy
Kurt Berckmueller, PhD, Fred Hutchinson Cancer
Research Center

4:45–5:00 PM 816: SARS-CoV-2 Spike Protein-Pseudotyped Lentiviral

Vectors (S-LV) for In Vitro and In Vivo Modelling of
Emergent SARS-CoV-2 Variants
Kamran Miah, PhD, MS, BS, University of Oxford

5:00–5:15 PM 817: A Single Injection of CD117 Antibody-drug Conjugate

Allows for Efficient Engraftment of Gene-modified CD34+
Cells in a Rhesus Gene Therapy Model
Naoya Uchida, MD, PhD, National Institutes of Health

5:15–5:30 PM 818: Bioinformatic-Guided Design of a Lentiviral

Vector for X-Linked Chronic Granulomatous Diseases
Recapitulates Endogenous CYBB Gene Regulation
and Expression
Ryan Wong, PhD, ImmunoVec

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WEDNESDAY, MAY 18, 2022

BALLROOM A
AAV Developments in Liver, T-cells, and Toxicity
Co-chairs: Nicole Paulk, PhD, UC San Francisco
and Amanda Dudek, PhD, Stanford University

3:45–4:00 PM 819: Alteration of ITR Sequences for Attenuating the

AAV Toxicity in Human Embryonic Stem Cells
Liujiang Song, PhD, University of North Carolina

4:00–4:15 PM 820: Preclinical Evaluation of AAV Vectors in an Ex Vivo

Human Whole Liver Explant Confirms the Potential of
Bioengineered AAVs as Clinically Relevant Hepatotropic
Vectors
ORAL ABSTRACT SESSIONS

Marti Cabanes-Creus, PhD, Children’s Medical Research

Institute

4:15–4:30 PM 821: Optimized Novel AAV Capsids Selected For Improved

Homology-Dependent Repair In Human T-cells
Adrian Westhaus, University College London

4:30–4:45 PM 822: Selection of Engineered AAV Capsids With Enhanced

Incorporation Into Extracellular Vesicles and Stable Liver
Transduction in vivo
Casey Maguire, PhD, The Massachusetts General Hospital

4:45–5:00 PM 823: Receptor and Antibody Interactions of AAV by

Cryo-EM and Tomography
Michael Chapman, PhD, University of Missouri

5:00–5:15 PM 824: Gene Expression From AAV Vectors in the Liver:

A Comparative Study Across Species, Promoters and
AAV Serotypes
Subha Karumuthil-Melethil, PhD, REGENXBIO Inc.

5:15–5:30 PM 825: Evolution of a New AAV Variant with Murine T

Lymphocyte Tropism using the MHC-Ib Molecule H2-Q7
as a Receptor
Jonathan Ark, Duke University

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HALL E
New Gene Editing Technologies and Applications
Co-chairs: Luca Biasco, PhD, AVROBIO and Paula Cannon, PhD, USC

3:45–4:00 PM 826: MitoTALENs as Genetic Tools to Reduce Mutant

mtDNA Levels in the CNS of a Mouse Model Carrying a
Heteroplasmic mtDNA Mutation
Sandra Bacman, PhD, University of Miami

4:00–4:15 PM 827: Lowering Huntington Gene Expression by

Targeted Base Editing of Cis-Regulatory Elements in
its Promoter Sequence
Tristan McCallister, University of Illinois Urbana Champaign
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 828: Long-read Nanopore Sequencing Reveals

Outcomes of AAV-CRISPR Editing in the Brain of
Transgenic Mouse Models
Bryan Simpson, The Children’s Hospital of Philadelphia

4:30–4:45 PM 829: Optimization of Pre-mRNA Exon Editing for Efficient

Rescue of ABCA4 Expression
Kirk Burkhart, PhD, Ascidian Therapeutics

4:45–5:00 PM 830: Promoterless AAV Vectors with Homology Arms can

Integrate and Express from Transcriptionally Active Sites
in Non-targeted Loci
Calvin Stephens, PhD, Stanford University

5:00–5:15 PM 831: SLEEK: A Method for Highly Efficient Knock-in and

Expression of Transgene Cargos for Next-generation
Cell-based Medicines
John Zuris, PhD, Editas Medicine

5:15–5:30 PM 832: Programmable Deletion, Replacement, Integration

and Inversion of Large DNA Sequences with Twin Prime
Editing and Site-specific Recombinases
Xin Gao, PhD, Broad Institute of Harvard and MIT

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ROOM 202
Novel Therapeutic Targets to treat CNS Disorders
Co-chairs: Miguel Sena-Esteves, PhD, UMass Chan Medical School
and Rebecca Ahrens-Nicklas, MD, PhD, Children’s Hospital of Philadelphia

3:45–4:00 PM 833: Hematopoietic Stem and Progenitor Cell Gene

Therapy Uniquely Benefits Multiple Sclerosis in the
Animal Model
Silvia Spadini, Division of Pediatric Hematology, Oncology
and Stem Cell Transplantation, Woman’s and Child Health
Department, University of Padova

4:00–4:15 PM 834: A Self-regulating Gene Therapy for Rett Syndrome

Stuart Cobb, PhD, Neurogene Inc.
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 835: A New Gene Therapy Approach to Treat

Niemann-Pick Type C2 Disease
Sara Marcó, PhD, Universitat Autònoma de Barcelona

4:30–4:45 PM 836: Comparison of Therapeutic Efficacy and Durability

of Gene Therapy for Tuberous Sclerosis Type 2 With
Standard of Care Everolimus
Edwina Abou Haidar
Massachusetts General Hospital

4:45–5:00 PM 837: A Novel Gene Therapy for Rett Syndrome Through

Reactivation of the Silent X Chromosome
Samantha Powers, PhD, Nationwide Children’s Hospital

5:00–5:15 PM 838: Second Generation AAV-mediated Gene

Therapy to Mitigate Risk for Alzheimer’s Disease in
APOE4/4Homozygotes
Rachel Montel, PhD, Weill Cornell Medicine

5:15–5:30 PM 839: Modulation of miR-181 Influences Dopaminergic

Neuronal Degeneration in a Mouse Model of
Parkinson’s Disease
Ryan Boudreau, PhD, University of Iowa

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ROOM 102
Harnessing Innate Immunity for Cancer Immunotherapy
Co-chairs: Gianpietro Dotti, MD, UNC Lineberger Cancer Center
and Sarwish Rafiq, PhD, Emory University

3:45–4:00 PM 840: Multiplex Base Editing of NK Cell to Enhance

Cancer Immunotherapy
Minjing Wang, University of Minnesota

4:00–4:15 PM 841: Engineered Induced Pluripotent Stem Cell-derived

Natural Killer Cells Reactively Co-target TIGIT and CD73
in the Glioblastoma Tumor Microenvironment
Kyle Lupo, Purdue University
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 842: Consistent Expansion and Activation of Autologous

Non-genetically Modified Natural Killer Cells With
Enhanced Cytotoxicity (SNK01) from Heavily Pre-treated
Patients With Advanced Solid Tumors
Paul Song, MD, NKGen

4:30–4:45 PM 843: Multifunctional Natural Killer Cell Engager Releasing

CXCL10 Augments Natural Killer Cell Recruitment and
Anti-tumor Efficacy Against Glioblastoma
Xue Yao, Purdue University

4:45–5:00 PM 844: Logic Gated FLT3 or CD33 Not EMCN CAR-NK

Cell Therapy (SENTI-202) for Precise Targeting of AML
Brian Garrison, PhD, Senti Biosciences

5:00–5:15 PM 845: FT536: A First-of-Kind, Off-the-Shelf CAR-iNK

Cell Product Candidate for Solid Tumors Designed to
Specifically Target MICA/B Stress Proteins and Overcome
Mechanisms of Tumor Evasion
John Goulding, PhD, Fate Therapeutics Inc.

5:15–5:30 PM 846: IFNalpha by In Vivo-engineered Macrophages

Abates Liver Metastases and Triggers Counter Regulatory
Responses Limiting Efficacy
Thomas Kerzel, SR-TIGET

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SALON G
Cell-based Cancer Immunotherapies II
Co-chairs: Masato Yamamoto, MD, PhD, University of Minnesota
and Irina Balyasnikova, PhD, Northwestern University Feinberg School of Medicine

3:45–4:00 PM 847: Characterization of the Transcriptomic and

T-cell Receptor (TCR) Clonal Heterogeneity of
Tumor-Infiltrating Lymphocyte (TIL) Therapy Infusion
Products by Single-Cell Sequencing and Correlative
Analyses With Clinical Efficacy in Patients With Advanced
Cutaneous Melanoma
Jinzhou Yuan, PhD, Instil Bio, Inc

4:00–4:15 PM 848: Dual CD33/CLL-1 Targeted CAR T-cells for Treatment

of Acute Myeloid Leukemia
ORAL ABSTRACT SESSIONS

Kevin Kowal, Washington University School of Medicine

4:15–4:30 PM 849: Off-the-shelf Natural Killer Cells Derived from HIPSC

via Genetic Modifications
Luhan Yang, PhD, Qihan Biotech

4:30–4:45 PM 850: Targeting the C Domain of Tenascin C with CAR

T-cells for the Immunotherapy of Pediatric Brain and
Solid Tumors
Elizabeth Wickman, St. Jude Graduate School

4:45–5:00 PM 851: A Phase 1 Dose Escalation Study of GCC19CART a

Novel CoupledCAR® Therapy for Subjects With Metastatic
Colorectal Cancer
Jiuwei Cui, MD, PhD, The First Bethune Hospital of
Jilin University

5:00–5:15 PM 852: Discovery of a Novel C07:02-Resticted Epitope on

MAGE-A1 and Pre-clinical Development of an Enhanced
TCR T-cell Therapy Candidate for the Treatment of
Solid Tumors
Gavin MacBeath, PhD, TScan Therapeutics

5:15–5:30 PM 853: Asymmetric cell division for fate induction of

chimeric antigen receptor (CAR) T-cells
Christoph Ellebrecht, MD, University of Pennsylvania

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WEDNESDAY, MAY 18, 2022

ROOM 206
Engineered Cell Therapies
Co-chairs: Bakhos Tannous, PhD, Harvard Medical School and
Massachusetts General Hospital
and M Graca Almeida-Porada, MD, PhD, Wake Forest School of Medicine

3:45–4:00 PM 854: Primary Human Hepatocytes, Genetically Engineered

ex vivo to be Hypoimmunogenic, Can Rescue a Model of
Metabolic Liver Disease
Fei Yi, Ambys Medicines

4:00–4:15 PM 855: Kinase p38 Regulates Macrophage Adaptation in the

Lung Upon Pulmonary Macrophage Transfer
Ariane Nguyen M. Sc., Hannover Medical School
ORAL ABSTRACT SESSIONS

4:15–4:30 PM 856: Ex Vivo Transduced Macrophages Engraft in the

Lung Following Transplantation and Produce Therapeutic
Levels of Secreted Proteins
Nora Clarke, PhD, Imperial College London

4:30–4:45 PM 857: Scalable Generation and Tailored Design of Human

iPSC-macrophages for Novel Immunotherapies Targeting
Bacterial Infections
Mania Ackermann, Hannover Medical School

4:45–5:00 PM 858: Bioprinting of Organotypic Aggregates of

Hepatocytes and Mesenchymal Cells as a Platform for
Liver Cell Therapy
Christopher Dickman, Aspect Biosystems

5:00–5:15 PM 859: iPSC-Derived Monocytes Generate Functional M1

and M2 Macrophages with Enhanced Cytokine Secretion
and Tumor Cell-Killing Activity
Ian Hay, Factor Bioscience

5:15–5:30 PM 860: Transplantation of Gene Edited Upper Airway

Basal Stem Cells in Immunocompromised Mice Using
Fibrinogen Based Scaffolds
Sriram Vaidyanathan, PhD, Stanford University

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ROOM 201
Vector Manufacturing and Engineering 3: Improving Vector Design
and System Performance
Chair: Christopher Doering, PhD, Emory University

3:45–4:00 PM 861: Size-Optimized and Shelf-Stable Plasmid DNA

Particles for Production of Viral Vectors
Yizong Hu, Johns Hopkins University

4:00–4:15 PM 862: The Primate Selective Transduction of rAAV-LK03

Vectors is Related to Variation in Histone and Histone
Post-translational Modifications on the Viral Genome in
the Host Nucleus
ORAL ABSTRACT SESSIONS

Adriana Gonzalez-Sandoval, PhD, Stanford University

4:15–4:30 PM 863: Development and Characterization of Highly

Optimized Monoclonal Producer Cell Lines (PCLs) for the
Treatment of CDKL5 Deficiency Disorder (CDD)
Laurie Tran, MSc, Ultragenyx Pharmaceutical

4:30–4:45 PM 864: Optimized Human Regulatory Sequences Achieve

Targeted Expression in CNS and Decreased Liver
Expression in Mice
Annie Tanenhaus, Encoded Therapeutics

4:45–5:00 PM 865: Development of Next Generation Vaccine Platform

with Self-amplifying mRNAs
Anitha Thomas, PhD PMP, Precision NanoSystems

5:00–5:15 PM 866: Long Term Stability Profiles of AAV Vectors at

Ambient Temperature within a Film Matrix
Maria Croyle, PhD, UT-Austin College of Pharmacy

5:15–5:30 PM 867: Combination of Advanced Plasmid Design,

Transfection Reagent and Design of Experiment (DOE)
Achieves High-yield, High-quality and Potent AAV Vectors
in Scalable Suspension HEK293 Cells
Jing Liao, PhD, LogicBio Therapeutics

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ROOM 204
Pharmacology/Toxicology Studies or Assay Development I
Co-chairs: Heikki Turunen, PhD, Dyno Therapeutics
and Eva Andres-Mateos, MD, PhD, Atsena Therapeutics S, Inc

3:45–4:00 PM 868: Circulating Neurofilament Light Chain as a Promising

Biomarker of AAV-induced Dorsal Root Ganglia Toxicity in
Nonclinical Toxicology Species
Kelly Fader, PhD, Pfizer Inc

4:00–4:15 PM 869: Development of Probe-Based qPCR Assays for the

Detection of Replication-Competent Lentiviral Particles
ORAL ABSTRACT SESSIONS

Menna Ahmed, ProtaGene CGT GmbH

4:15–4:30 PM 870: Sonication Linker Mediated-PCR (SLiM-PCR), an

Efficient Method for Quantitative Retrieval of Vector
Integration Sites
Fabrizio Benedicenti, SR-TIGET

4:30–4:45 PM 871: CDMS Analysis of DNA Released From rAAV Gene

Therapy Vectors
Benjamin Draper, PhD, Megadalton Solutions

4:45–5:00 PM 872: The Safety and Biodistribution Profiles of

Systemically Delivered Oncolytic Adenovirus in Pigs
Margarita Romanenko, PhD, University of Minnesota

5:00–5:15 PM 873: Electrophysiology and Soluble Biomarker as

Translational Tools to Monitor Adeno-Associated-Virus
Related Ganglionopathy
Juliette Hordeaux, University of Pennsylvania

5:15–5:30 PM 874: Application of Single-Cell Transcriptomics to Assess

rAAV-Delivered Transgene Expression
Richard Lamontagne, University of Pennsylvania

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HALL D
Networking Reception & Poster Session III
5:30–6:30 PM

INTERNATIONAL SPY MUSEUM

Networking Event—Closing Night Reception
8:00–11:00 PM

Join us for a night of fun during the Closing Night Reception at Washington,
D.C.’s International Spy Museum! Enjoy the museum exhibits, light appetizers,
and drinks.
You can purchase a ticket ($50 per person) for the reception before or after you
register for the meeting. Space is limited!

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 The one-time-only dose to stop SMA progression
ZOLGENSMA is a gene therapy for pediatric patients less than 2 years
of age with spinal muscular atrophy (SMA), that is delivered as a single-
dose, 1-hour intravenous infusion1

91% (20/22) of patients were alive and free of permanent

Event-free ventilation at the 14-months-of-age study visit, a primary
survival endpoint, and at 18 months of age2,a-c

59% (13/22) of patients achieved the ability to sit without support

Motor for ≥30 seconds at the 18-month study visit, a primary endpoint2,a
milestones
86% (19/22) of patients achieved one or more motor milestones
achieved by 18 months of age2,a

Rapid As early as 1 month post infusion, CHOP INTEND scores

onset increased from baseline by a mean of 6.9 points (N=22)2,a

The efficacy of ZOLGENSMA was evaluated in STR1VE, a completed, open-label, single-arm,

multicenter, Phase 3 clinical trial of patients with SMA Type 1 (genetically confirmed bi-allelic SMN1
deletion, 2 copies of SMN2, and <6 months of age at symptom onset and treatment; N=22).1,a,b

Get started with ZOLGENSMA today:

Call 1-855-441-GENE (4363) or learn more at
[Link]

a
One patient was initially classified as presymptomatic and removed from the intent-to-treat (ITT) data set included in the Prescribing Information. The patient was later
confirmed to be symptomatic at baseline and included in the final ITT analysis. 2
b
One patient died at age 7.8 months due to respiratory failure, which was considered unrelated to treatment. One patient withdrew consent at 11.9 months of age; this patient
required permanent ventilation at 11.0 months prior to withdrawal of consent. One patient discontinued participation at the age of 18.0 months, before the month 18 end-of-
study visit, due to an adverse event of respiratory distress, which was considered unrelated to treatment.2
c
Event is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days in the absence of
an acute reversible illness, excluding perioperative ventilation.1
d
Treated globally in clinical trials, managed access programs, and commercially.

Indication and Important liver impairment may be at higher risk. Prior blood count before ZOLGENSMA infusion.
to infusion, assess liver function of all patients Following infusion, monitor for thrombocytopenia as
Safety Information by clinical examination and laboratory well as other signs and symptoms of TMA. Consult a
Indication testing (e.g., hepatic aminotransferases pediatric hematologist and/or pediatric nephrologist
ZOLGENSMA is an adeno-associated virus vector- [aspartate aminotransferase (AST) and alanine immediately to manage if clinically indicated.
based gene therapy indicated for the treatment of aminotransferase (ALT)], total bilirubin, and
pediatric patients less than 2 years of age with spinal prothrombin time). Administer a systemic Elevated Troponin-I
muscular atrophy (SMA) with bi-allelic mutations in corticosteroid to all patients before and after Increases in cardiac troponin-I levels were observed
the survival motor neuron 1 (SMN1) gene. ZOLGENSMA infusion. Continue to monitor liver following ZOLGENSMA infusion. Monitor troponin-I
Limitations of Use function for at least 3 months after infusion. before ZOLGENSMA infusion and on a regular basis
The safety and effectiveness of repeat administration WARNINGS AND PRECAUTIONS for at least 3 months afterwards.
or the use in patients with advanced SMA (e.g., Thrombocytopenia ADVERSE REACTIONS
complete paralysis of limbs, permanent ventilator Transient decreases in platelet counts, some of The most commonly observed adverse reactions
dependence) has not been evaluated with which met the criteria for thrombocytopenia, were (incidence ≥5%) in clinical studies were elevated
ZOLGENSMA. typically observed within the first two weeks after aminotransferases and vomiting.
ZOLGENSMA infusion. Monitor platelet counts before
Important Safety Information ZOLGENSMA infusion and on a regular basis for at Please see Brief Summary of Prescribing
BOXED WARNING: Acute Serious least 3 months afterwards. Information on the adjacent page.
Liver Injury and Acute Liver Failure Thrombotic Microangiopathy References: 1. ZOLGENSMA [prescribing information].
Acute serious liver injury, acute liver failure, Cases of thrombotic microangiopathy (TMA) were Bannockburn, IL: Novartis Gene Therapies, Inc; 2021.
and elevated aminotransferases can occur reported approximately 1 week after ZOLGENSMA 2. Data on file. AveXis, Inc. 2020. 3. Data on file.
with ZOLGENSMA. Patients with preexisting infusion. Obtain baseline creatinine and complete Novartis Gene Therapies, Inc. 2022.

© 2022 Novartis Gene Therapies, Inc. For US healthcare professionals only.

Bannockburn, IL 60015 03/2022 US-ZOL-22-0047
ZOLGENSMA® (onasemnogene abeparvovec-xioi) Rx Only a pediatric hematologist and/or pediatric nephrologist immediately to manage TMA
Suspension for intravenous infusion as clinically indicated.
Brief Summary of the Full Prescribing Information. Elevated Troponin-I
See Full Prescribing Information. Increases in cardiac troponin-I levels (up to 0.176 mcg/L) were observed following
ZOLGENSMA infusion in clinical trials. The clinical importance of these findings is not
BOXED WARNING: ACUTE SERIOUS LIVER INJURY known. However, cardiac toxicity was observed in animal studies. Monitor troponin-I
AND ACUTE LIVER FAILURE before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards
(weekly for the first month, and then monthly for the second and third months until
• Acute serious liver injur y, acute liver failure, and elevated troponin-I level returns to baseline). Consider consultation with a cardiologist, if
aminotransferases can occur with ZOLGENSMA. troponin elevations are accompanied by clinical signs or symptoms.
• Patients with preexisting liver impairment may be at higher risk. ADVERSE REACTIONS
• Prior to infusion, assess liver function of all patients by clinical The safety data described in this section reflect exposure to ZOLGENSMA in four
examination and laboratory testing (e.g., hepatic aminotransferases open-label studies conducted in the United States, including one completed clinical
[aspartate aminotransferase (AST) and alanine aminotransferase trial, two ongoing clinical trials, and one ongoing observational long-term follow-up
(ALT)], total bilirubin, and prothrombin time). Administer systemic study of the completed trial. A total of 44 patients with SMA received intravenous
corticosteroid to all patients before and after ZOLGENSMA infusion. infusion of ZOLGENSMA, 41 patients at or above the recommended dose, and
Continue to monitor liver function for at least 3 months after infusion. 3 patients at a lower dose. The patient population ranged in age from 0.3 months
to 7.9 months at the time of infusion (weight range 3.0 kg to 8.4 kg). The most
INDICATIONS AND USAGE frequent adverse reactions (incidence ≥ 5%) observed in the 4 studies were elevated
aminotransferases* 27.3% (12/44) and vomiting 6.8% (3/44).
ZOLGENSMA is an adeno-associated virus vector-based gene therapy indicated
*Elevated aminotransferases include elevation of alanine aminotransferase (ALT) and/or aspartate
for the treatment of pediatric patients less than 2 years of age with spinal muscular aminotransferase (AST). In the completed clinical trial, one patient (the first patient infused in that
atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. study) was enrolled prior to the protocol amendment instituting administration of prednisolone
Limitation of Use: The safety and effectiveness of repeat administration of before and after ZOLGENSMA infusion.
ZOLGENSMA or the use in patients with advanced SMA (e.g., complete paralysis of One patient in an ongoing non-United States clinical trial initially presented with
limbs, permanent ventilator dependence) has not been evaluated. respiratory insufficiency 12 days after ZOLGENSMA infusion and was found to have
DOSAGE AND ADMINISTRATION respiratory syncytial virus (RSV) and parainfluenza in respiratory secretions. The
patient had episodes of serious hypotension, followed by seizures, and was found
For single-dose intravenous infusion only. to have leukoencephalopathy (brain white matter defects) approximately 30 days
The recommended dosage of ZOLGENSMA is 1.1 × 10 14 vector genomes per after ZOLGENSMA infusion. The patient died after withdrawal of life support 52 days
kilogram (vg/kg) of body weight. after ZOLGENSMA infusion.
• Administer ZOLGENSMA as an intravenous infusion over 60 minutes. Immunogenicity
• Postpone ZOLGENSMA in patients with concurrent infections until the infection has In ZOLGENSMA clinical trials, patients were required to have baseline anti-AAV9
resolved. Clinical signs or symptoms of infection should not be evident at the time antibody titers of ≤ 1:50, measured using an enzyme-linked immunosorbent assay
of ZOLGENSMA administration. (ELISA). Evidence of prior exposure to AAV9 was uncommon. The safety and efficacy
• Starting one day prior to ZOLGENSMA infusion, administer systemic corticosteroids of ZOLGENSMA in patients with anti-AAV9 antibody titers above 1:50 have not been
equivalent to oral prednisolone at 1 mg/kg of body weight per day for a total of evaluated. Perform baseline testing for the presence of anti-AAV9 antibodies prior
30 days. At the end of the 30-day period of systemic corticosteroid treatment, to ZOLGENSMA infusion. Retesting may be performed if anti-AAV9 antibody titers
check liver function by clinical examination and by laboratory testing. For are reported as > 1:50.
patients with unremarkable findings, taper the corticosteroid dose gradually Following ZOLGENSMA infusion, increases from baseline in anti-AAV9 antibody titers
over the next 28 days. If liver function abnormalities persist, continue systemic occurred in all patients. In the completed clinical trial, anti-AAV9 antibody titers reached
corticosteroids (equivalent to oral prednisolone at 1 mg/kg/day) until findings at least 1:102,400 in every patient, and titers exceeded 1:819,200 in most patients.
become unremarkable, and then taper the corticosteroid dose gradually over the Re-administration of ZOLGENSMA in the presence of high anti-AAV9 antibody titer
next 28 days or longer if needed. Do not stop systemic corticosteroids abruptly. If has not been evaluated.
liver function abnormalities continue to persist ≥ 2 × ULN after the 30-day period
DRUG INTERACTIONS
of systemic corticosteroids, consult a pediatric gastroenterologist or hepatologist.
Where feasible, adjust a patient’s vaccination schedule to accommodate concomitant
WARNINGS AND PRECAUTIONS corticosteroid administration prior to and following ZOLGENSMA infusion.
Acute Serious Liver Injury, Acute Liver Failure or Elevated Aminotransferases Certain vaccines, such as MMR and varicella, are contraindicated for patients on
Acute serious liver injury, acute liver failure and elevated aminotransferases a substantially immunosuppressive steroid dose (i.e., ≥ 2 weeks of daily receipt of
can occur with ZOLGENSMA. Hepatotoxicity (which may be immune-mediated), 20 mg or 2 mg/kg body weight of prednisone or equivalent). Seasonal RSV
generally manifested as elevated ALT and/or AST levels and at times as acute prophylaxis is not precluded.
serious liver injury or acute liver failure, has been reported with ZOLGENSMA USE IN SPECIAL POPULATIONS
use. In order to mitigate potential aminotransferase elevations, administer Pediatric Use
systemic corticosteroid to all patients before and after ZOLGENSMA infusion.
Immune-mediated hepatotoxicity may require adjustment of the corticosteroid Administration of ZOLGENSMA to premature neonates before reaching full-term
treatment regimen, including longer duration, increased dose, or prolongation gestational age is not recommended, because concomitant treatment with
of the corticosteroid. Patients with preexisting liver impairment or acute hepatic corticosteroids may adversely affect neurological development. Delay ZOLGENSMA
viral infection may be at higher risk of acute serious liver injury/acute liver failure. infusion until the corresponding full-term gestational age is reached. There is no
Patients with ALT, AST, or total bilirubin levels (except due to neonatal jaundice) information on whether breastfeeding should be restricted in mothers who may
> 2 × ULN have not been studied in clinical trials with ZOLGENSMA. The risks and be seropositive for anti-AAV9 antibodies. The safety of ZOLGENSMA was studied
benefits of infusion with ZOLGENSMA in patients with preexisting liver impairment in pediatric patients who received ZOLGENSMA infusion at age 0.3 to 7.9 months
should be weighed carefully against the risks of not treating the patient. Although in (weight range 3.0 kg to 8.4 kg). The efficacy of ZOLGENSMA was studied in pediatric
the clinical trials and in postmarketing experience, asymptomatic aminotransferase patients who received ZOLGENSMA infusion at age 0.5 to 7.9 months (weight range
elevations were very commonly reported, in the managed access program and 3.6 kg to 8.4 kg).
in the postmarketing setting, cases of acute serious liver injury and acute liver Hepatic Impairment
failure have been reported. Some patients have experienced elevations in ALT ZOLGENSMA therapy should be carefully considered in patients with liver impairment.
and AST > 20 × ULN, prolonged prothrombin time and have been symptomatic Cases of acute serious liver injury and acute liver failure have been reported with
(e.g., vomiting, jaundice), which resolved with the use of prednisolone, sometimes ZOLGENSMA in patients with preexisting liver abnormalities. In clinical trials, elevation
requiring prolonged duration and/or a higher dose. If acute serious liver injury or of aminotransferases was observed in patients following ZOLGENSMA infusion.
acute liver failure is suspected, consult a pediatric gastroenterologist or hepatologist.
Prior to ZOLGENSMA infusion, assess liver function by clinical examination and PATIENT COUNSELING INFORMATION
laboratory testing (hepatic aminotransferases [AST and ALT], total bilirubin level, See the ZOLGENSMA Full Prescribing Information for the Patient Counseling
and prothrombin time). Continue to monitor liver function for at least 3 months after Information.
ZOLGENSMA infusion (weekly for the first month, and then every other week for the Please visit [Link] for Full Prescribing Information,
second and third months, until results are unremarkable). including Boxed Warning.
Thrombocytopenia
Transient decreases in platelet counts, some of which met the criteria for
thrombocytopenia, were typically observed within the first two weeks after Manufactured by, Packed by, Distributed by:
ZOLGENSMA infusion. Monitor platelet counts before ZOLGENSMA infusion and on a Novartis Gene Therapies, Inc.
regular basis afterwards (weekly for the first month; every other week for the second 2275 Half Day Road, Suite 200
and third months until platelet counts return to baseline). Bannockburn, IL 60015 USA
Thrombotic Microangiopathy
U.S. License No. 2250
Cases of thrombotic microangiopathy (TMA) were reported approximately one week
after ZOLGENSMA infusion in the post-marketing setting. TMA is characterized by
thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney injury.
Concurrent immune system activation (e.g., infections, vaccinations) was identified
in some cases. Monitor platelet counts, as well as signs and symptoms of TMA, such as
hypertension, increased bruising, seizures, or decreased urine output. In case these
signs and symptoms occur in the presence of thrombocytopenia, further diagnostic
evaluation for hemolytic anemia and renal dysfunction should be undertaken. If © 2021 Novartis Gene Therapies, Inc.
clinical signs, symptoms and/or laboratory findings consistent with TMA occur, consult Bannockburn, IL 60015 11/2021 US-ZOL-19-0202 V4
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THURSDAY, MAY 19, 2022

7:00 AM–12:00 PM REGISTRATION HOURS—EAST SALON

ROOM 201
Effective Regulatory Interactions
Co-chairs: Megan Zoschg-Canniere, PhD, Spark Therapeutics
and Kit Shaw, PhD, Dana-Farber Cancer Institute

8:00–8:10 AM ASGCT Survey on Sponsor Communication Challenges

Megan Zoschg-Canniere, PhD, Spark Therapeutics

8:10–8:25 AM FDA-Sponsor Communications

SCIENTIFIC SYMPOSIA

Wilson Bryan, MD, Food and Drug Administration

8:25–8:40 AM Key Proposals Within PDUFA to Address Communication

Challenges
Khushboo Sharma, PhD, Biotechnology Innovation
Organization

8:40–8:55 AM Optimizing Early FDA Interactions (Pre-IND)

Aron Stein, PhD, Intellia Therapeutics

8:55–9:10 AM Strategies for Effective Late-stage

FDA Communications
Anita Freed, PhD, Pfizer

9:10–9:45 AM Panel Discussion

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BALLROOM B
Emerging Clinical and Translational Safety Topics in Cell and Gene Therapy
Co-Chair: Megan Hoban, PhD, bluebird bio and TK

8:00–8:25 AM Advanced Vectors for In Vivo CAR T-cell Generation

SCIENTIFIC SYMPOSIUM

Christian J. Buchholz, PhD, Paul Erhlich Institut, Germany

8:25–8:50 AM MDS/AML After Curative Therapies for

Sickle Cell Disease
Courtney Fitzhugh, MD, National Institutes of Health

8:50–9:15 AM Product-derived Lymphoma Following Infusion

of PiggyBac Modified CD19 Chimeric Antigen
Receptor T-cells
Kenneth Micklethwaite, MD, PhD, Westmead Hospital

9:15–9:40 AM Roadmap for Determining Vector Involvement

in Hematologic Malignancy
Melissa Bonner, PhD, bluebird bio

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BALLROOM A
Finding a “Cure”: The Promise and Perils of Gene Therapy,
In Utero and Beyond
Co-chairs: Randy J. Chandler, PhD, National Institutes of Health
and Stephanie Cherqui, PhD, UC San Diego

8:00–8:25 AM In Utero Gene Editing for Congenital Diseases

William Peranteau, MD, Children’s Hospital of Philadelphia
SCIENTIFIC SYMPOSIUM

8:25–8:50 AM Gene-based Therapeutics Operating Under

Natural Regulation and Targeting Common Causes
of Rare Diseases
Dan Wang, PhD, University of Massachusetts
Chan Medical School

8:50–9:15 AM HSC Gene Therapy Applications in Neurometabolic and

Neurodegenerative Disorders
Alessandra Biffi, MD, DFCI/Boston Children’s Cancer
and Blood Disorders Center

9:15–9:40 AM Xona Drug-induced Splicing Switch for Regulated Control

of Gene Therapies
Alejandro Mas Monteys, PhD, University of Philadelphia;
Children’s Hospital of Philadelphia

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ROOM 207
Global Experience With COVID-19 Vaccines
Co-chairs: Shan Lu, MD, PhD, University of Massachusetts Chan Medical School
and David B. Weiner, PhD, Wistar Institute

8:00–8:25 AM A Gene-based Vaccine Platform Leads to

Durable Immunity
Luk Vandenberghe, PhD, ciendias bio, Grousbeck Gene
Therapy Center, Mass Eye and Ear, Harvard & The Broad
Institute of Harvard and MIT

8:25–8:50 AM Duration of Immunity, Long-term Prospects

to Control COVID
Alessandro Sette, [Link], La Jolla Institute for
Immunology
SCIENTIFIC SYMPOSIA

8:50–9:15 AM Prospect for Universal COVID Vaccine

Steven L. Zeichner, MD, PhD, University of Virginia

9:15–9:40 AM Community-acquired Immunity Against SARS-CoV-2

Prevents Hospitalization, Severe Disease and Death
Against Delta and Omicron Variants Better Than Vaccine-
induced Immunity—At a High Cost for Many
Gary P. Kobinger, PhD, Galveston National Laboratory (GNL),
UT Medical Branch at Galveston

SALON G
What are the Most Pressing Issues of the Future?
Chair: Rayne Rouce, MD, Baylor College of Medicine

8:00–9:45 AM Panel Discussion

Sarwish Rafiq, PhD, Emory University
Li Ou, PhD, Capsida Biotherapeutics
Alisha Gruntman, DVM, PhD, Tufts Cummings School of
Veterinary Medicine; University of Massachusetts Chan
Medical School
Avery D. Posey, Jr, PhD University of Pennsylvania Perelman
School of Medicine

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ROOM 204
Cutting Edge Gene and Cell Therapy Research in Europe,
in Memory of Dr. Manfred Schmidt
Co-chairs: Juan A. Bueren, PhD, CIEMAT
and Hildegard Büning, PhD, Hannover Medical School

8:00–8:26 AM Manfred’s Legacy: Assaying the Pharmacodynamics

of Insertional Gene Therapy
Christof von Kalle, PhD, Berlin Institute of Health (Charite)
SCIENTIFIC SYMPOSIA

8:26–8:52 AM How Integration Site Analysis Impacted the Design

of Safer Vectors and Clinical Trials
Axel Schambach, MD, PhD, Hannover Medical School

8:52–9:18 AM DNA Integration in Therapeutic Gene Modification:

History and Current Challenges
Frederic D. Bushman, PhD, University of Pennsylvania
School of Medicine

9:18–9:44 AM Next-generation Genotoxicity Testing and

Clonal Tracking Methodologies
Eugenio Montini, PhD. San Raffaele Telethon Institute
for Gene Therapy

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BALLROOM C
Clinical Trials Spotlight Symposium
Chair: Maria Grazia Roncarolo, MD, Stanford University

8:00–8:15 AM 1188: Interim Results from an Ongoing Phase 1/2 Study

of Lentiviral-Mediated Ex Vivo Gene Therapy for Pediatric
Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Donald B. Kohn, MD, UCLA

8:15–8:30 AM 1189: Safety and Efficacy of SARS-COV-2-specific T-cells

as Adoptive Immunotherapy for High-risk COVID-19
Patients: A Phase I/II, Randomized Clinical Trial
Evangelia Yannaki, George Papanikolaou Hospital

8:30–8:45 AM 1190: Autologous Cell & Gene Therapy for the Therapeutic
ORAL ABSTRACT SESSIONS

Targeting of Immune Payloads to the Solid Tumor

Microenvironment: Preliminary Results of the TEM-GBM Study
Bernhard Gentner, MD, PhD, SR-TIGET

8:45–9:00 AM 1191: Lentiviral Gene Therapy with Low Dose Busulfan

for Infants With X-Linked Severe Combined Immune
Deficiency (XSCID) results in the Development of a
Normal and Sustained Immune System: Interim Results of
an Ongoing Phase I/II Clinical Study
Ewelina Mamcarz, MD, St. Jude Children’s Research Hospital

9:00–9:15 AM 1192: Stable Hemostatic Correction and Improved

Hemophilia-Related Quality of Life: Final Analysis From
the Pivotal Phase 3 HOPE-B Trial of Etranacogene
Dezaparvovec
Steven Pipe, University of Michigan

9:15–9:30 AM 1193: IGNITE DMD Study of SGT-001 Microdystrophin

Gene Therapy for Duchenne Muscular Dystrophy:
Long-Term Outcomes and Biomarker Update
Carl Morris, PhD, Solid Biosciences

9:30–9:45 AM 1194: The OPTIC Study of Intravitreal Gene Therapy With

ADVM-022 for Neovascular AMD (nAMD): the Role of
Neutralizing Antibodies
Szilard Kiss, MD, Weill Cornell Medical College

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BALLROOM A
Novel AAV Capsids for the Brain, Eye and Kidney
Co-chairs: Nicole Paulk, PhD, UC San Francisco
and Andrew Steinsapir, Deerfield Management

10:15–10:30 AM 1195: Machine-Guided Design Reveals AAV Variants

Efficient in Transducing NHP Retina After
Intravitreal Delivery
Heikki Turunen, Dyno Therapeutics

10:30–10:45 AM 1196: A Newly Evolved AAV Variant Enables Potent

Gene Transfer in Kidneys of Multiple Species
Alan Rosales, BS, MS, Duke University
ORAL ABSTRACT SESSIONS

10:45–11:00 AM 1197: A Promising New Family of Peptide-modified AAV

Capsids for Gene Delivery to the Central Nervous System
in Non-human Primates
Ken Chan, PhD, Broad Institute of MIT and Harvard

11:00–11:15 AM 1198: Directed Evolution of AAV9 Peptide Display Libraries

Identifies a Family of Cross-Species Variants With
Enhanced Brain Tropism in Non-Human Primates and
Mice Following Systemic Administration
Tyler Moyer, PhD, Voyager Therapeutics

11:15–11:30 AM 1199: Novel AAV Capsids for Intravitreal Delivery:

Identifying and Characterizing Novel AAV Variants in
Non-Human Primates
Karen Guerin, PhD, Vedere Bio II

11:30–11:45 AM 1200: Identification and Characterization of an AAV9-

Based Engineered Capsid Variant Capable of Mediating
Enhanced Transcription in the Central Nervous System of
Non-Human Primates and Rodents
April Giles , PhD, REGENXBIO

11:45 AM–12:00 PM 1201: Fit4Function: A Machine Learning-guided Approach

for Systematic Multi-trait AAV Capsid Engineering
Fatma-Elzahraa Eid, PhD, Broad Institute of MIT and Harvard

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BALLROOM C
New Technologies for AAV Gene Therapy
Co-chairs: Phillip Tai, PhD, UMass Chan Medical School
and Aravind Asokan, PhD, Duke University School of Medicine

10:15–10:30 AM 1202: Effects of Complement Component 1 (C1) Inhibition

on AAV-based Gene Transfer Efficacy and Immunogenicity
in Mice
Nikki Ross, PhD, Spark Therapeutics

10:30–10:45 AM 1203: Inducible Gene Expression for Gene Therapy:

Design and Exemplification of Powerful, Small, Modular
and Tightly Controlled Regulatable Promoters
Graham Whyteside, PhD, Asklepios Biopharmaceutical Inc
ORAL ABSTRACT SESSIONS

10:45–11:00 AM 1204: Inclusion of AAV Empty Capsids can Increase

Expression in Certain Contexts In Vitro and In Vivo
Julie Crudele, PhD, University of Washington

11:00–11:15 AM 1205: Disease-responsive Therapy for the Treatment of

Myotonic Dystrophy
Ellie Carrell, PhD, Children’s Hospital of Philadelphia

11:15–11:30 AM 1206: Preclinical Gene Reactivation of the X-linked

Cdkl5 Gene using Dual Adeno-associated Virus Mediated
CRISPR/dCas9 Epigenetic Editing
Julian Halmai, PhD, MS, UC Davis

11:30–11:45 AM 1207: Characterization of Optimized Dual AAV-MYO7A

Vectors for the Treatment of Usher Syndrome (USH1B) in
Myo7a-/- Mice and NHP
Kaitlyn Calabro, PhD, University of Florida

11:45 AM–12:00 PM 1208: Differential Histopathological and Proteomics

Changes in Dorsal Root Ganglia (DRG) of Cynomolgus
Macaques Following Intrathecal (IT) Delivery of “Empty”
AAV9 Capsids, and AAV9 carrying Transcriptionally Active
or Inert Cargo
Ghiabe Guibinga, PhD, Novartis Institutes for BioMedical
Research (NIBR)

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BALLROOM B
AAV Vectors—Clinical Studies
Co-chairs: Kathryn Wagner, MD, PhD, F. Hoffmann-La Roche
and Christian Mueller, PhD, Sanofi Genzyme

10:15–10:30 AM 1209: A Phase 2 Trial Evaluating Safety and Efficacy

of Delandistrogene Moxeparvovec in Duchenne
Muscular Dystrophy
Teji Singh, MD, PhD, Sarepta Therapeutics, Inc.

10:30–10:45 AM 1210: Evaluation of Total Binding Antibodies Against

rAAVrh74 in Patients with Duchenne Muscular Dystrophy
ORAL ABSTRACT SESSIONS

Natalie Goedeker, CPNP, Washington University School

of Medicine

10:45–11:00 AM 1211: Phase 1 Study of Gene Therapy in Late-onset

Pompe Disease: Initial 104-Week Experience
Edward Smith, MD, Duke University School of Medicine

11:00–11:15 AM 1212: Sustained Efficacy and Safety at Week 52 and up

to Three Years in Adults With Glycogen Storage Disease
Type iA (GSDIa): Results From a Phase 1/2 Clinical Trial of
DTX401, an AAV8-mediated, Liver-directed Gene Therapy
Rebecca Riba-Wolman, MD, University of Connecticut

11:15–11:30 AM 1213: Rationally Designed Cardiotropic AAV Capsid

Demonstrates 30 Fold Higher Efficiency in Human vs
Porcine Heart
Tugba Guven-Ozkan, PhD, AskBio Biopharmaceutical

11:30–11:45 AM 1214: Safety, β-Sarcoglycan Expression, and Functional

Outcomes From Systemic Gene Transfer of rAAVrh74.
[Link] in LGMD2E/R4
Eric Pozsgai, Sarepta Therapeutics, Inc.

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ROOM 202
Delivery Technologies and In Vivo Gene Editing
Co-chairs: TJ Cradick, PhD, Excision BioTherapeutics, Inc
and Daniela Cesana, PhD, SR-TIGET

10:15–10:30 AM 1215: Durable Silencing of Pcsk9 by In Vivo

Hit-and-run Epigenome Editing
Martino Alfredo Cappelluti, IRCCS San Raffaele
Scientific Institute

10:30–10:45 AM 1216: AAV-CRISPR/Cas9 Gene Editing is Therapeutic in a

Novel, Humanized Mouse Model of GUCY2D-associated
ORAL ABSTRACT SESSIONS

Cone Rod Dystrophy (CORD6)

Russell Mellen, PhD, University of Florida

10:45–11:00 AM 1217: A Large Scale Exon Editing Solution for Treating

ABCA4 Related Retinopathies
Robert Bell, PhD, Ascidian Therapeutics

11:00–11:15 AM 1218: Highly Efficient and Safe In Vivo HSC Base Editing
for the Treatment of Hemoglobinopathies
Chang Li, PhD, University of Washington

11:15–11:30 AM 1219: Dual Editing and in vivo Selection at the

HBG Promoter Using MGMT-P140K Cassette
Mason Berger, Seattle Children’s Research Institute

11:30–11:45 AM 1220: In Vivo Expansion Of Gene-targeted Hepatocytes

Through Inhibition Of An Essential Gene
Marco De Giorgi, PhD, Baylor College of Medicine

11:45 AM–12:00 PM 1221: Intravitreal Delivery of AAV2 - Exon-specific

U1snRNA corrects ELP1 Splicing and Rescues Retinal
Degeneration in a Mouse Model of Familial Dysautonomia
Anil Chekuri, PhD, Massachusetts Eye and Ear Infirmary

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ROOM 201
Breakthroughs in Neuromuscular and Hearing Disorders
Co-chairs: Scott Q. Harper, PhD, Ohio State University
and Rachel M. Bailey, PhD, UT Southwestern

10:15–10:30 AM 1222: CYP46A1 as a Relavant Target to Treat

ALS Pathology Independent From its Origin
Françoise PIGUET, PhD, Neurogencell, ICM

10:30–10:45 AM 1223: Gene Therapy for ALS by Specifically

Overexpressing a Pleiotropic Chronokine,
Secreted α-Klotho, in Skeletal Muscles
Sergi Verdés, PhD Student
ORAL ABSTRACT SESSIONS

10:45–11:00 AM 1224: AAV-9 Mediated Delivery of RNA Targeting Systems

Eliminates Hexanucleotide Repeat Expansions in C9ORF72
ALS/FTD Models
Jeannie Chew, PhD, Locanabio

11:00–11:15 AM 1225: TFRC-Targeted GAA Delivered as Gene Therapy

Treats CNS and Muscle in Pompe Disease Model Mice
Maria Praggastis, PhD, Regeneron

11:15–11:30 AM 1226: Preclinical Development of an AAV-based Gene

Therapy (OTO-825) for Congenital Hearing Loss Due to
GJB2 Deficiency
Phillip Uribe, PhD, Otonomy

11:30–11:45 AM 1227: Adult AAV Gene Therapy Rescues Auditory Function

in a Mouse Model of Human TMPRSS3 Recessive Deafness
DFNB8/10
Wan Du, MD, PhD, Harvard Medical School/Mass Eye
and Ear

11:45 AM–12:00 PM 1228: A Knockdown and Replacement Strategy for the

Treatment of Charcot Marie Tooth Type 2A
Eileen Workman, University of Pennsylvania

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SALON G
Ophthalmic and Auditory Diseases
Co-chairs: Hemant Khanna, PhD, IVERIC Bio and Mariacarmela Allocca, PhD,
Editas Medicine

10:15–10:30 AM 1229: A Mutation-Independent CRISPR/Cas9-based

‘Knockout and Replace’ Strategy to Treat Rhodopsin-
Associated Autosomal Dominant Retinitis Pigmentosa
Chi-Hsiu Liu, PhD, Editas Medicine, Inc.

10:30–10:45 AM 1230: Development of an AAV-based Gene Therapy for the

Ocular Phenotype of Friedreich’s Ataxia
Siddhant Gupte, University of Florida
ORAL ABSTRACT SESSIONS

10:45–11:00 AM 1231: SPVN06, a Novel Mutation-independent AAV-based

Gene Therapy, Dramatically Reduces Vision Loss in the rd10
Mouse Model of Rod-cone Dystrophy and is Well Tolerated
in a 1-month Pilot Safety Monkey Study
Florence Lorget, SparingVision

11:00–11:15 AM 1232: Reduced Ocular Inflammation and Improved GFP

Expression in Rabbits with Controlled Release of Adeno-
Associated Virus from Degradable Hydrogel Implants
Steven Lu, PhD, Ocular Therapeutix

11:15–11:30 AM 1233: Nonclinical In Vivo Expression, Durability of Effect,

Biodistribution/Shedding, and Safety Evaluations Support
Clinical Development of AK-OTOF (AAVAnc80-hOTOF
Vector) for OTOF-mediated Hearing Loss
Ann Hickox, PhD, Akouos, Inc.

11:30–11:45 AM 1234: Novel Capsid LSV1 has a Unique 3D Structure at

the Loop Substitution Area - Confers Superior Retinal
Transduction from Intravitreal Injection
Cameron Baker, PhD, Adverum

11:45 AM–12:00 PM 1235: Development of Dual-PCDH15 AAV Gene Therapy for

Usher Syndrome Type 1F Deafness and Blindness
Maryna Ivanchenko, PhD, MD, Harvard Medical School

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ROOM 207
Cell-Based Cancer Immunotherapies III
Co-chairs: Saad Kenderian, MD, Mayo Clinic
and Michael Milone, MD, PhD, University of Pennsylvania

10:15–10:30 AM 1236: Optimized CD70-Targeted CAR Secreting a

CD33-Targeted Bispecific T-cell Engager Overcomes
Antigen Heterogeneity for Acute Myeloid Leukemia
Mark Leick, MD, Massachusetts General Hospital
Cancer Center

10:30–10:45 AM 1237: Safety Lead-in of Ph2 AML Study Results Using

Zedenoleucel
Mythili Koneru, MD, PhD, Marker Therapeutics
ORAL ABSTRACT SESSIONS

10:45–11:00 AM 1238: N-glycosylation Inhibition Hinders

Immunosuppressive Activity of Tumor Microenvironment
Cells and Improves CAR T Cell Efficacy
Camilla Sirini, San Raffaele Scientific Institute

11:00–11:15 AM 1239: CAR T That Targets MUC1 Transmembrane Cleavage

Product has Increased Persistence and Kills Low Antigen Cells
Cynthia Bamdad, PhD, Minerva Biotechnologies

11:15–11:30 AM 1240: CD4 CAR T Cells Drive Extensive CD8 CAR T-cell
Expansion, Leading to Severe Cytokine Release Syndrome
Camilla Bove, Innovative Immunotherapies Unit, IRCCS
San Raffaele Scientific Institute

11:30–11:45 AM 1241: Targeting Tumors and the Tumor Microenvironment

with Banana Lectin Expressing T-cells
Mary Kathryn K. McKenna, PhD, Baylor College of Medicine

11:45 AM–12:00 PM 1242: Preclinical Activity and Safety of UB-VV100, A Novel

Lentiviral Vector Product Designed for Selective and
Effective In Vivo Engineering of Therapeutic Anti-CD19
CART Cells for B-Cell Malignancies
Alissa Brandes, PhD, Umoja Biopharma

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ROOM 204
Cell Therapies for Hematological Disorders
Co-chairs: Andre Larochelle, MD, PhD, NIH
and Punam Malik, MD, PhD, Cincinnati Children’s Hospital Medical Center

10:15–10:30 AM 1243: Ex Vivo Generated ProTcellTM Product Exhibits Fate

Plasticity Between T and NK Lineages: An Opportunity for
Innate and Adaptive Cell Therapy Strategies
Pierre Gaudeaux, MS, Smart Immune

10:30–10:45 AM 1244: Hypoimmunemouse Primary Pancreatic Islet Cells

Survive and Functionally Rescue Allogeneic Diabetic Mic
Sonja Schrepfer, MD, PhD, Sana Biotechnology Inc

10:45–11:00 AM 1245: FVIII-Expressing Human Placental Cells Engraft in

ORAL ABSTRACT SESSIONS

Multiple Organs and Provide FVIII Protein While Evading

Induction of FVIII Inhibitors When Administered to
Juvenile Sheep
Brady Trevisan, Wake Forest Institute for
Regenerative Medicine

11:00–11:15 AM 1246: Modeling Clinical Scale, Efficacious CRISPR-edited

HSPC Therapies in Nonhuman Primates
Jason Murray, PhD, Fred Hutchinson Cancer Research Center

11:15–11:30 AM 1247: Engineering T-cells To Prevent Acute Graft-Versus-

Host Disease And Leukemia Relapse Following Allogeneic
Stem Cell Transplantation
Feiyan Mo, Baylor College of Medicine

11:30–11:45 AM 1248: Postnatal Boosting With FVIII-Expressing Human

Placental Cells Supports Immune Tolerance was Induced
During Prenatal Exposure, and is BTLA-Mediated
Martin Rodriguez, BS, Wake Forest Institute for
Regenerative Medicine

11:45 AM–12:00 PM 1249: Diphtheria Toxin Based Bivalent Anti-cMPL

Immunotoxin Effectively and Safely Depletes Rhesus
Hematopoietic Stem and Progenitor Cells
Daisuke Araki, Cellular and Molecular Therapeutics Branch,
NHLBI, NIH

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SCHEDULE (ALL TIMES LISTED IN ET)
THURSDAY, MAY 19, 2022

ROOM 206
Cell Therapy Product Engineering, Development or Manufacturing
Co-chairs: Isabelle Riviere, PhD, Memorial Sloan-Kettering Cancer Center
and Maksim Mamonkin, PhD, Baylor College of Medicine

10:15–10:30 AM 1250: Improved Anti-tumor Potency of CAR Gamma Delta

T-cells Expanded with Human Platelet Lysate
Norihiro Watanabe, PhD, Baylor College of Medicine

10:30–10:45 AM 1251: Base Editors as a Singular Platform for

Polyfunctional Multiplex Engineering of T-cells for
Cancer Immunotherapy
ORAL ABSTRACT SESSIONS

Walker Lahr, University of Minnesota

10:45–11:00 AM 1252: Allogeneic CAR T Cells Derived From Younger

Donor T Cells Have More Desirable T-cell Phenotype and
Better In Vitro Functionality
Meng-Yin Lin, Allogene Therapeutics

11:00–11:15 AM 1253: Enhanced CAR T-cell Generation by CD8-LV

Through Alleviating Antiviral Mechanisms with
mTOR Inhibitors
Filippos Charitidis, MSc, Paul Ehrlich Institut

11:15–11:30 AM 1254: Generation of Off-the-shelf Allogeneic

Hypoimmune Tregs
Ivayla Gyurova, PhD, Sana Biotechnology

11:30–11:45 AM 1255: Dysfunctional Immune Synapses Restrain Anti DIPG

Activity of CAR T-cells
Jorge Ibanez, PhD, St. Jude Children’s Research Hospital

11:45 AM–12:00 PM 1256: mRNA-based Gene Editing in Primary Human

Muscle Stem Cells
Christian Stadelmann, Muscle Research Unit, Experimental
and Clinical Research Center,

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SCHEDULE (ALL TIMES LISTED IN ET)
THURSDAY, MAY 19, 2022

ROOM 102
Pharmacology/Toxicology Studies or Assay Development II
Co-chairs: Angela Lynch, PhD, ToxPlus Consulting, LLC
and Eva Andres-Mateos, PhD, Atsena Therapeutics S, Inc

10:15–10:30 AM 1257: A GLP-compliant Toxicology and Biodistribution

Study of ADVM-062 (AAV.7m8-L-opsin), a Novel Gene
Therapy Product Being Developed as a Potential Single
Intravitreal Administration for the Treatment of Blue Cone
Monochromacy
Kelly Hanna, MS, Adverum Biotechnologies

10:30–10:45 AM 1258: Visium CytAssist: A Novel Platform for Spatial

Transcriptomic Analysis of FFPE Sections Mounted on
Standard Glass Slides
ORAL ABSTRACT SESSIONS

Hardeep Singh, Senior Scientist, 10x Genomics

10:45–11:00 AM 1259: Development of an AAV-Based Gene Therapy for

Children With Congenital Hearing Loss Due to Otoferlin
Deficiency (DB-OTO)
Orion Keifer Jr, MD, PhD, Decibel Therapetics

11:00–11:15 AM 1260: Long-read Sequencing and Multiplex ddPCR for

Viral Vector Genome Integrity Identification
David Dobnik, PhD, National Institute of Biology

11:15–11:30 AM 1261: Toxicity of Frataxin Overexpression in Nonhuman

Primates Treated with Intravenous and MRI-guided
Intracerebellar Infusion of an AAV Vector
Christian Hinderer, University of Pennsylvania

11:30–11:45 AM 1262: ASC618, a Second Generation of FVIII Gene Therapy

for Hemophilia A, Exhibits Major Transduction and
Transgene Expression in the Target Liver Tissues: Results
of IND-enabling Pharmacokinetics Studies in Mice and
Non-Human Primates
Chengtao Yang, ASC Therapeutics

11:45 AM–12:00 PM 1263: Safety and Biodistribution of VTX-801, an AAV3B

Gene Therapy Vector, in Healthy Cynomolgus Monkeys
Blanche Tamarit, Vivet Therapeutics

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ABSTRACT REVIEWERS
A1 - RNA Virus Vectors
Review Chair: Patricia Devaux, PhD – Mayo Clinic
Alessandra Biffi, MD – DFCI/BCH Cancer and Blood Disorders Center
Douglas Jolly, PhD – Abintus Bio Inc.
John Tisdale, MD – NIH, NHLBI
Andrew Wilber, PhD – Southern Illinois University School of Medicine
Mario Amendola, PhD – Genethon

A2 - AAV Vectors—Virology and Vectorology

Review Chair: Nicole Paulk, PhD – UC San Francisco
Daniel Lipinski, MSc,DPhil – Medical College of Wisconsin
Anna Maurer, PhD – UC Berkeley
Amanda Dudek, PhD – Stanford University
Rafael Yáñez-Muñoz, PhD – Royal Holloway University of London
Isabelle Richard, PhD – Genethon

A3 - AAV Vectors—Preclinical and Proof-of-Concept

Studies
Review Chair: Heather Gray-Edwards, DVM, PhD – UMass Chan Medical
School
Brian Bigge, PhD – University of Manchester
Philip Tai, PhD – UMass Chan Medical School
Allison Bradbury, PhD – Nationwide Children’s Hospital
Juliette Hordeaux , DVM, PhD, DECVP – University of Pennsylvania
Rita Batista, PhD – UMass Chan Medical School

A4 - AAV Vectors—Clinical Studies

Review Chair: Kathryn Wagner, MD, PhD – F. Hoffmann-La Roche
Carolyn Lutzko, PhD – Cincinnati Childrens Hospital Medical Center
Katherine High, MD – Ask Bio
Chris Mueller, PhD – Sanofi Genzyme
Ana Buj Bello, MD, PhD – Genethon
Gallia Levy, MD, PhD – Spark Therapeutics

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B - Gene Targeting and Gene Correction
Review Chair: Maria Grazia Roncarolo, MD – Stanford University
Paula Cannon, PhD – University of Southern California
TJ Cradick, PhD – Excision BioTherapeutics
Mara Pavel-Dinu, PhD – Stanford University
Giuliana Ferrari, PhD – HSR-TIGET, Fondazione S. Raffaele
Luca Biasco, PhD – AVROBIO

C - Oligonucleotide Therapeutics
Review Chair: Paloma Giangrande, PhD – Wave Life Sciences
Marcin Kortylewski, PhD – City of Hope Comprehensive Cancer Center
Michelle Hastings, PhD – Rosalind Franklin University
Janaiah Kota, PhD – Indiana University School of Medicine
Shen Shen, PhD – Vertex Pharmaceuticals
Mark Behlke, MD, PhD – IDT

D - Synthetic/Molecular Conjugates and Physical

Methods for Delivery of Gene Therapeutics
Review Chair: Carol Miao, PhD – Seattle Children’s Research Institute
Dexi Liu, PhD – University of Georgia
Julie Champion (Synthetic), PhD – Georgia Tech School of Chemical
and Biomolecular Engineering
Kenya Kamimura (Physical), MD, PhD – Niigata University
Richard Heller (Physical), PhD – University of South Florida
David Dean (Physical), PhD – University of Rochester
Fan Yuan (Synthetic), PhD – Duke University

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E1 - Metabolic, Storage, Endocrine, Liver and
Gastrointestinal Diseases
Review Chair: Nicola Brunetti-Pierri, MD – Telethon Institute of Genetics
and Medicine
Gloria Gonzalez-Aseguinolaza, PhD – Vivet Therapeutics
Charles Venditti, MD, PhD – NIH
Stephanie Cherqui, PhD – UC San Diego
Gerald Lipshutz, MD – David Geffen School of Medicine at UCLA
Andres Muro, PhD – International Centre for Genetic Engineering and
Biotechnology (ICGEB)

E2 - Cardiovascular and Pulmonary Diseases

Review Chair: Uta Griesenbach, PhD – Imperial College Faculty
of Medicine
Kate Excoffon (Respiratory), PhD – Spirovant Sciences, Inc.
Patrick Sinn, PhD – University of Iowa
Margaret Sleeper (Cardio), VMD, DACVIM – University of Florida
Mai ElMallah, MD – Duke University Medical School
Gerry McLachlan, PhD – University of Edinburgh

E3 - Neurologic Diseases
Review Chair: Allison Bradbury – Ohio State University
Kathrin Meyer, PhD – Nationwide Children’s Hospital
Miguel Sena-Esteves, PhD – UMass Chan Medical School
Kleopas Kleopa, MD, PhD – Cyprus Institute of Neurology and Genetics
Laura Ferraiuolo, PhD – Sheffield University
Gad Vatine, PhD – University of Negev
Rebecca Ahrens-Nicklas, MD, PhD – Children’s Hospital of Philadelphia

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E4 - Ophthalmic and Auditory Diseases
Review Chair: Luk Vandenberghe, PhD – Harvard University
Litia Carvalho, PhD – MGH
Deniz Dalkara, PhD – Institute de lab vision, Paris
Hemant Khanna, PhD – University of Massachusetts
Yen-Fu Cheng, MD, PhD – Veterans Hospital Taipei
Casey Maguire, PhD – Massachusetts General Hospital

E5 - Musculo-skeletal Diseases
Review Chair: Dongsheng Duan, PhD – University of Missouri School
of Medicine
Annemieke Aartsma-Rus, PhD – Leiden University Medical Center
Bruce Smith, VMD, PhD – Auburn University
Louise Rodino-Klapac, PhD – Sarepta Therapeutics

E6 - Cancer - Immunotherapy, Cancer Vaccines

Review Chair: Chiara Bonini, MD – Universita Vita Salute San Raffaele
Monica Casucci, PhD – San Raffaele Scientific Institute
Ann Leen, PhD – Baylor College of Medicine - CAGT
Barbara Savoldo, MD, PhD – UNC Lineberger Comprehensive
Cancer Center
Michael Hudecek, MD – University Hospital Wurzburg
Sarwish Rafiq, PhD – Emory University

E7 - Cancer—Oncolytic Viruses
Review Chair: Melissa Kotterman, PhD – 4D Molecular Therapeutics
(4DMT)
Marta Alonso, PhD – University Hospital of Navarra
Evanthia Galanis, MD – Mayo Clinic
Deepak Verma, PhD – Johns Hopkins University
Paola Grandi, PhD, MBA – CG Oncology
Sunil S. Raikar, MD – Emory University

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E8 - Cancer—Targeted Gene and Cell Therapy
Review Chair: Michael Milone, MD, PhD – University of Pennsylvania
Masato Yamamoto, MD, PhD – University of Minnesota
Daniel Abate-Daga, PhD H – Lee Moffitt Cancer Center and
Research Institute
Barbra Sasu, PhD – Allogene
Saad Kenderian, MB, ChB – Mayo Clinic
Irina Balyasnikova, PhD – Northwestern University Feinberg School
of Medicine

E9 - Hematologic and Immunologic Diseases

Review Chair: Juan Bueren, PhD – CIEMAT/CIBERER/Fundacion
Jiminez Diaz
Bernhard Gentner, MD, PhD – SR-TIGET
Donald Kohn, MD – UCLA
Anne Galy, PhD – GENETHON
Claire Booth, MBBS, PhD – UCL Great Ormond Street Institute of
Child Health

F - Immunological Aspects of Gene Therapy and Vaccines

Review Chair: Allison Keeler-Klunk, PhD – UMass Chan Medical School
Julie Crudele, PhD – University of Washington
Gwladys Gernoux, PhD – Nantes University
Chengwen Li, MD, PhD – UNC Chapel Hill
Oumeya Adjali, MD, PhD – INSERM U1089
Ying Kai Chan, PhD – Harvard University

G - Cell Therapies
Review Chair: Punam Malik, MD, PhD – Cincinnati Childrens Hospital
Medical Center
Grace Almeida-Porada, MD, PhD – Wake Forest of Medicine
Andre Larochelle, MD, PhD – NIH
Jan Nolta, PhD – UC Davis
Alice Tarantal, PhD – UC Davis
Stefano Rivella, PhD – Children’s Hospital of Philadelphia
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H1 - Vector Product Engineering, Development
or Manufacturing
Review Chair: H. Trent Spencer, PhD – Emory University School
of Medicine
Johannes van der Loo, PhD – Children’s Hospital of Philadelphia
John Gray, PhD – Vertex Pharmaceuticals
Anindya Dasgupta, PhD – Expression Manufacturing, LLC
Kerry Dooriss, PhD – City of Hope Comprehensive Cancer Center

H2 - Cell Therapy Product Engineering, Development

or Manufacturing
Review Chair: Adrian Gee, PhD – Baylor College of Medicine
Maksim Mamonkin, PhD – Baylor College of Medicine
Daniela Bischof, PhD – Indiana University School of Medicine
Katy Rezvani, MD, PhD – MD Anderson Cancer Center
Isabelle Riviere, PhD – Memorial Sloan-Kettering Cancer Center
Edwin Horwitz, MD, PhD – Emory University School of Medicine

H3 - Pharmacology/Toxicology Studies or Assay

Development
Review Chair: Eva Andres-Mateos, PhD – Atsena Therapeutics, Inc.
Angela Lynch, PhD – ToxPlus Consulting, LLC
Sarah Wassmer, PhD – Gene Therapy Directorate
Heikki Turunen, PhD – Dyno Therapeutics
Linda Couto, PhD – Atsena Therapeutics, Inc.
Caner Gunaydin, PhD

I - Gene and Cell Therapy Trials in Progress

Review Chair: Kevin Flanigan, MD – Nationwide Children’s Hospital
Diana Bharucha-Goebel, MD – NIH/NINDS and Children’s
National Hospital
Barry J. Byrne, MD, PhD – University of Florida
Helen E. Heslop, MD, DSc – Baylor College of Medicine
Carsten Bonnemann, MD – NINDS/NIH

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