Our ability to treat diseases previously untreatable through the delivery of adeno-associated virus (AAV)-based gene therapies has been a tremendous advancement in pharmaceutical development. The ability to bring promising products in the pipeline to the clinic and ultimately to the market is hampered by inefficiencies in the development of the manufacturing processes and analytical tools, and challenges with scalability. This is largely attributable to the biological complexity of the products, the optimization needed every time a new AAV serotype capsid vector is being developed for gene therapy, and limited understanding of the structure-function relationship that guides optimization of the manufacturing process and analytical testing

The diseases that necessitate the implementation of gene therapy to treat patients are often characterized by small patient populations. The number of these diseases is quite extensive leaving pharma companies faced with completing the extensive set of CMC requirements needed to support a regulatory filing for each new product. This is labor intensive and costly, often outstripping the ability of companies to recover the costs of bringing these products to market.

A platform approach can be developed for a class of products (e.g., AAV vectors, lentivirus vectors) after manufacturers have gained considerable experience, and have developed a production strategy based on similar manufacturing processes/unit operations. Platform-based approaches have been used successfully to streamline manufacturing of well characterized biologics like mAbs to reduce the CMC burden and cost.

The goal of the session is to further our understanding of how manufacturers have streamlined the development of AAV-based gene therapy products through the utilization of platform approaches. The session will include presentations from developers and CDMOs that have adopted platform approaches to decrease the CMC burden and bring promising therapies through the pipeline (in early development) to the clinic and through late phase development and commercialization to patients. The objective of the session is to share experiences, strategies and regulatory feedback that facilitated the development of platform approaches for AAV based gene therapie

Session Speakers:

Balancing Flexibility and Standardization of AAV Manufacturing Processes

Mercedes Segura, ElevateBio

Gaining Momentum from an AAV Gene Therapy Manufacturing Platform

Joe Peltier, BioMarin Pharamceutical Inc.

Shoring up Your Platform: Applying Knowledge Management and Regulatory Considerations to Gene Therapy Development

Shannon Holmes, Ultragenyx Pharmaceutical Inc.

Additional Panelists:

Andrew Harmon, CBER, FDA

Zenobia Taraporewala

Appearances