FDA + ASGCT

Immune Responses to AAV Vectors

January 25, 2023 | 10:00 AM CT


Already Registered? Watch this event on-demand!

Watch Day One

Watch Day Two

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

Speakers & Topics

Jan. 24, 2023

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

Jan. 25, 2023

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

2024

Register for the 27th Annual Meeting

May 7-11, 2024 | Baltimore, MD

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