Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data

Watch Day 1 of this event!

Welcome & Introduction

Immunogenicity in AAV Gene Therapy Products: Identifying Areas That May Benefit from Convergence

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
FDA perspective on immunogenicity, including assay validation and regulatory considerations
- Speaker: Andrew Byrnes, PhD, FDA OTP
Panel Discussion
- Moderator: Terence Flotte, MD, UMass Chan Medical School
- Additional participants:
  - Kirsten Coleman, University of Florida
  - Megha Kaushal, MD, FDA OTP

Integration of Clinical and Preclinical Data: Making Preclinical Models More Predictive of Clinical Observed Toxicity

FDA Perspective on Data Integration - How clinical data informs pre-clinical study design and regulatory implications.
- Speaker: Margaret Benny Klimek, PhD, FDA OTP
Down-translating human clinical data to preclinical models and selecting appropriate models.
- Speaker: Allison Keeler, PhD, UMass Chan Medical School
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

Break

Challenges in Animal Models: Overcoming Resource Constraints, Long Timelines, and Limitations of Models

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.
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