FDA + ASGCT

Immune Responses to AAV Vectors

January 25, 2023 | 10:00 AM CT

Topics will include, but are not limited to:

Requirements for immunogenicity testing, including data that should be collected and what may be relied upon from previous applications, and recommendations on clinically meaningful metrics
Appropriateness and utility of a companion diagnostic for assessing pre-existing immunity and requirements for product approval

11 a.m. – 3 p.m. (ET)

Registrants & members: Watch Day 1 On Demand

11-11:15 a.m.
Federico Mingozzi, PhD (Spark Therapeutics)
Welcome & introduction
11:15-11:45 a.m.
Shari Gordon, PhD (AskBio)
Overview of immunogenicity and clinical trials with healthy volunteers
11:45 a.m.-12:15 p.m.
J. Fraser Wright, PhD (Stanford University)
Vector process and manufacturing: How does it impact immunogenicity?
12:15-12:45 p.m.
Manuela Corti, PhD (University of Florida)
Immunomodulation as an adjunctive therapy to AAV systemic dosing to improve safety and allow for repeated AAV dosing
12:45-1:15 p.m.
Natasha Thorne, PhD (FDA)
Regulating companion diagnostics for AAV-based gene therapies
1:45-2:45 p.m.
Panel: Shari Gordon, J. Fraser Wright, Manuela Corti, Natasha Thorne, and Lei Xu, MD, PhD (OTAT, CBER, FDA)
Panel discussion

11 a.m. – 3 p.m. (ET)

Registrants & members: Watch Day 2 On Demand

11-11:15 a.m.
Ying Kai Chan, PhD (Harvard)
Welcome & introduction
11:15-11:45 a.m.
Genevieve Laforet, MD, PhD (Aspa Therapeutics)
Complement-Mediated Thrombotic Microangiopathy associated with AAV gene therapy
11:45 a.m.-12:15 p.m.
Carsten Bonnemann, MD (NINDS/NIH)
AAV gene therapy related immune responses: Selected clinical observations from the GAN, DMD, and MTM1 trials
12:15-12:45 p.m.
Terry Flotte, MD (UMass Chan Medical School)
rAAV Gene Therapy for Tay-Sachs and Sandhoff Diseases: Focus on Immune Responses
12:45-1:15 p.m.
Dave Copland, PhD (University of Bristol)
Intraocular AAV inflammation
1:45-2:45 p.m.
Panel: Genevieve LaForet, Terry Flotte, Dave Copland, Carsten Bonnemann
Panel discussion