With the recent approval of several Adeno-Associated Virus (AAV)-based gene therapies, the application of AAV gene therapy approaches to the treatment of human disease is expanding. AAV is emerging as the vector of choice for gene therapy based on a developing understanding of numerous features of its utility, including, but not limited to its’ safety, versatility, durability, and manufacturing approaches.

As this knowledge base expands, a given AAV quality target product profile (i.e., specific AAV serotype for targeting a specific organ or cell types) may lend itself to being the basis for a platform approach to product development for targeting specific monogenic diseases impacting similar target organs or cell types.

The advantages to this approach can be to leverage prior knowledge from preclinical studies and manufacturing process development, including an understanding of product critical quality attributes, critical process parameters for process steps, including those assessed for virus clearance, analytical methods and other elements of the control strategy, to create synergies for other products in development. This synergistic platform approach to AAV development is essential to help reduce costs, streamline development, and ultimately deliver these products to the patients who need them more efficiently.

This session will highlight different approaches taken by industry, CMOs and other organizations and illustrate their experience, including the benefits achieved with the application of platform approaches to AAV product development.

Session Speakers:

Establishing Analytical Tools for an AAV Platform

Melissa Clague, Eli Lilly and Company

Assembling Complex Lego Blocks - Building AAV Manufacturing Platforms

John Kerwin, National Resilience, Inc.

NIH Programs Supporting the Development of AAV as a Therapeutic Platform

PJ Brooks, NCATS, NIH

Additional Panelists:

Christiane Niederlaender, Parexel International

Allison Wolf

Appearances

Parallel Session 10: Platform Development Approaches for AAV Gene Therapy Products

Session Chairs

Speakers