Agenda Details | ASGCT Annual Meeting

Detailed Program

Tuesday, May 07 2024
08:00 AM – 12:00 PM ET
Ballroom 3

AAV Manufacturing for CMC Success: Translating Preclinical Gene Therapy Products to Clinical Success

This workshop is especially geared to those who are early-career or who want to learn the basics of CMC considerations for AAV gene therapies. The intended audience for this workshop includes those working in early-stage companies (pre-Phase I) developing AAV gene therapy products.

The AAV manufacturing workshop will provide an overview of how early process development decisions for an AAV drug product impacts IND enabling GLP studies that will ultimately inform success in the GMP manufacturing environment for clinical trials, and eventually a BLA. Importantly, the drug product is comprised of: i) the AAV capsid to ensure delivery to a specific tissue/cellular location, while de-targeting others, and ii) the genetic payload constituting the therapeutic of interest. Early decisions during the research and development phase around choice of capsid and design of the genetic payload can influence design of the CMC process and the biological outcome associated with the drug product. The first half of the workshop will review how early decisions influence CMC manufacturing from an academic and industry perspective, the importance of appropriate analytics to analyze the drug product and designing IND enabling toxicology studies that most closely emulate human biological outcomes. The second half of the workshop will provide a regulatory perspective on CMC, review the practicalities for moving CMC to novel AAV production platforms, and provide insights around the CMC impacts of applying A.I. to early design of the drug product.

Session Details

08:00 AM - 12:00 PM - Ballroom 3

Adam Cockrell - PhD - Solid Biosciences

Co-Chair

08:00 AM - 12:00 PM - Ballroom 3

Nathalie Clement - PhD - Siren Biotechnology

Co-Chair

08:00 AM - 08:05 AM - Ballroom 3

Nathalie Clement - PhD - Siren Biotechnology

Welcome & Introduction

08:05 AM - 08:25 AM - Ballroom 3

Johannes van der Loo - PhD - Children's Hospital of Philadelphia

AAV Manufacturing now and then: the role of academia in shaping today’s industry platforms.

08:25 AM - 08:45 AM - Ballroom 3

David Dismuke - Forge Biologics

Preparing for CMC success: how early CMC decision drives your pace and success to the clinic

08:45 AM - 09:05 AM - Ballroom 3

Kirsten Coleman - MBA - University of Florida

How to design phase-appropriate successful IND-enabling tox studies for an AAV DP

09:05 AM - 09:40 AM - Ballroom 3

Kirsten Coleman - University of Florida

Panelist

09:05 AM - 09:40 AM - Ballroom 3

David Dismuke - Forge Biologics

Panelist

09:05 AM - 09:40 AM - Ballroom 3

Johannes Van Der Loo - PhD - Children's Hospital of Philadelphia

Panelist

10:00 AM - 10:20 AM - Ballroom 3

Bo Liang - FDA CBER OTAT

CMC considerations for adeno-associated virus (AAV)-vector based gene therapies - a CBER perspective

10:20 AM - 10:40 AM - Ballroom 3

Matthias Hebben - Complement Therapeutics

Successful manufacturing platforms and strategies to support clinical development up to BLA filing

10:40 AM - 11:00 AM - Ballroom 3

Lauren Kelly - MS - Siren Biotechnology

The future: Moving toxicology studies from the animal to in vitro analysis

11:00 AM - 11:20 AM - Ballroom 3

Claire Aldridge - PhD -

Using A.I. to shape early design of the drug product and the manufacturing process

11:20 AM - 11:55 AM - Ballroom 3

Matthias Hebben - Complement Therapeutics

Panelist

11:20 AM - 11:55 AM - Ballroom 3

Lauren Kelly -

Panelist

11:20 AM - 11:55 AM - Ballroom 3

Claire Aldridge - PhD -

Panelist

11:20 AM - 11:45 AM - Ballroom 3

Bo Liang - FDA CBER OTAT