ASGCT and FDA will host a virtual scientific workshop over two half days on January 22 and 23, 2025 (9a.m. - 1 p.m. ET on both days). This free, public event will explore key immunology-related topics that have implications across the class of AAV gene therapies, including immune responses, antibody testing, and redosing strategies.
The workshop will provide valuable insights for professionals in both academia and industry as it addresses pressing challenges in AAV gene therapies and new approaches.
Additionally, attendees will gain important regulatory perspectives from the FDA, offering guidance on how these ongoing issues are being addressed within the regulatory landscape.
Agenda
Wednesday January 22, 2025
9 - 9:05 a.m.
Welcome & Introduction
9:05 - 10:20 a.m.
Immunogenicity in AAV Gene Therapy Products: Identifying Areas That May Benefit from Convergence
- 9:05-9:25 a.m.
Consistency around assays for detecting preexisting immunity, including binding/neutralizing antibody cutoffs. Maternal antibody tracking and novel studies aimed at reducing immunogenicity.
- Speaker: Terence Flotte, MD, UMass Chan Medical School
- 9:25-9:45 a.m
FDA perspective on immunogenicity, including assay validation and regulatory considerations
- Speaker: Andrew Byrnes, PhD, FDA OTP
- 9:45-10:20 a.m.
Panel Discussion
- Moderator: Terence Flotte, MD, UMass Chan Medical School
- Additional participants:
- Kirsten Coleman, University of Florida
- Megha Kaushal, MD, FDA OTP
10:20 - 11:35 a.m.
Integration of Clinical and Preclinical Data: Making Preclinical Models More Predictive of Clinical Observed Toxicity
- 10:20-10:40 a.m.
FDA Perspective on Data Integration - How clinical data informs pre-clinical study design and regulatory implications.
- Speaker: Margaret Benny Klimek, PhD, FDA OTP
- 10:40-11:00 a.m.
Down-translating human clinical data to preclinical models and selecting appropriate models.
- Speaker: Allison Keeler, PhD, UMass Chan Medical School
- 11:00-11:35 a.m.
Panel Discussion
- Moderator: Melissa Spencer, PhD, UCLA
- Additional participants:
- Barry Byrne, MD, PhD, University of Florida
- Laura Hagerty, PhD, ReveraGen BioPharma, Inc.
- Upendra (Upen) Mahat, MD, FDA OTP
11:35 - 11:45 a.m.
Break
11:45 a.m. - 1 p.m.
Challenges in Animal Models: Overcoming Resource Constraints, Long Timelines, and Limitations of Models
- 11:45 a.m.-12:05 p.m.
Challenges in using animal models for AAV research, including the shortage of NHPs, differences in immune responses, and potential new approaches.
- Speaker: Juliette Hordeaux, DVM, PhD, GEMMA Biotherapeutics, Inc.
- 12:05-12:25 p.m.
Considerations for animal models for AAV development: An FDA Perspective
- Speaker: David Cantu, PhD, FDA OTP
- 12:25-1:00 p.m.
Panel Discussion
- Moderator: Emmanuel Adu-Gyamfi, PhD, Bristol-Myers Squibb, Inc.
- Additional participants:
- David Markusic, PhD, Spark Therapeutics, Inc.
- Aravind Asokan, PhD, Duke University School of Medicine
Thursday January 23, 2025
9 - 9:05 a.m.
Welcome
9:05 - 10:20 a.m.
Antibody Testing and Companion Diagnostics: Avoiding Duplication of Efforts When Possible
- 9:05-9:25 a.m.
FDA perspective on considerations for antibody testing and companion diagnostics in AAV therapies.
- Speaker: Johnny Lam, PhD, FDA OTP
- 9:25-10:20 a.m.
Panel Discussion
- Moderator: Barry Byrne, MD, PhD, University of Florida
- Additional Participants:
- Joanne Lemmo, Sarepta Therapeutics, Inc.
- Emily Coonrod, PhD, ARUP Laboratories, Inc.
10:20 - 11:35 a.m.
Redosing Strategies in AAV Gene Therapies: Overcoming Neutralizing Antibodies
- 10:20-10:40 a.m.
Challenges and strategies for redosing, especially in seropositive patients.
- Speaker: Barry Byrne, MD, PhD, University of Florida
- 10:40-11:00 a.m.
Discussion on the challenges and strategies for redosing, especially in seropositive patients. This session will highlight the use of Imlifidase as a potential approach to enable AAV redosing, with insights from Sarepta.
- Speaker: Louise Rodino-Klapac, PhD, Sarepta Therapeutics, Inc.
- 11:00-11:35 a.m.
Panel Discussion
- Moderator: Aravind Asokan, PhD, Duke University School of Medicine
- Additional participants: Charles Askew, PhD, NabGen, Inc.
11:35 - 11:45 a.m.
Break
11:45 a.m. - 1 p.m.
Immunosuppression in AAV Gene Therapies: What Does the Clinical Evidence Support?
- 11:45 a.m.-12:05 p.m.
Variability of immunosuppression approaches, including the use of corticosteroids and phase-appropriate strategies.
- Speaker: Kevin Flanigan, MD, Nationwide Children’s Hospital
- 12:05-12:25 p.m.
FDA Perspective on regulatory considerations for immunosuppression, focusing on clinical phase appropriateness.
- Speaker: Rosa Sherafat-Kazemzadeh, MD, FDA OTP
- 12:25-1:00 p.m. - Panel Discussion
- Moderator: Barry Byrne, MD, PhD, University of Florida
- Additional participants:
- Cynthia Tifft, MD, PhD, NIH
- Jonathon Schwartz, MD, Rocket Pharmaceuticals, Inc.
Attendee and Speaker Social Media Resources
We encourage attendees and speakers alike to use social media to maximize your presence at the upcoming ASGCT-FDA workshop! Below are a few example images and a downloadable document of social media posts that you may use to inform your audience about your participation at Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data!