Error loading player: No playable sources found

89823

Plenary Session 7 - Streamlining AAV Vector-based Gene Therapy Development Through the Utilization of Manufacturing and Analytical Platform Approaches

Date
June 13, 2024
This product is not available for individual purchase, but it is available as part of the following products:

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 PeltierBioMarin Pharamceutical Inc.


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

Shannon HolmesUltragenyx Pharmaceutical Inc.



Additional Panelists:


Andrew HarmonCBER, FDA


Related Products

Thumbnail for Plenary Session 8 - Global Regulatory Updates and Panel Discussion & Closing Remarks
Plenary Session 8 - Global Regulatory Updates and Panel Discussion & Closing Remarks
The cell and gene therapy field spans an enormous range of product types and technologies with the potential to provide curative treatments for a wide range of serious diseases…
Thumbnail for Plenary Session 4 - Potency Assays for Gene Edited Products
Plenary Session 4 - Potency Assays for Gene Edited Products
Measurement of the biological activity is crucial for lot release, stability and any comparability exercise of all biological medicinal products…
Thumbnail for Plenary Session 6 - Fireside Chat: Evolution of Cell and Gene Therapy
Plenary Session 6 - Fireside Chat: Evolution of Cell and Gene Therapy
Join us for an engaging fireside chat delving into the revolutionary landscape of cell and gene therapy…
Thumbnail for Plenary Session 1 - Genetic Modification Technologies for Cell-based Gene Therapies
Plenary Session 1 - Genetic Modification Technologies for Cell-based Gene Therapies
In addition to the now well-known CAR T (chimeric antigen receptor T cells) and HSC (CD34+ hematopoietic stem cells) cell-based gene therapies, there are several other modes of gene modification as well as cell types already approved and on the horizon…