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64137
Parallel Session 1: CMC Challenges with Ultra Rare Diseases
Date
June 27, 2023
This product is not available for individual purchase, but it is available as part of the following products:
The Cell and Gene Therapy Products (CGTP) Symposium: Manufacturing, Quality and Regulatory Considerations enables the exchange of scientific ideas and dialogue with regulators that form the basis of evolving regulatory practices in the development of these diverse and innovative products…
Bringing treatment to those afflicted with an ultra-rare disease is complex. These products are encumbered with challenges ranging from high development costs with a limited ROI, to issues with executing and interpreting clinical trials with unusually small patient populations. Furthermore, the cost to generate the quantities of product required to support an approvable CMC package can be prohibitive for many organizations.
This session will address ways in which companies have tackled the CMC and regulatory challenges when bringing these life changing therapies to patients in need.
Session Speakers:
Manufacturing Challenges Limiting the Access to ATMPs
Ralf Altenburger, F. Hoffmann-La Roche Ltd.
Development of a Single Patient CRISPR Therapeutic
Richard Horgan, Cure Rare Disease
Additional Panelists:
Emmanuel Adu-Gyamfi, CBER, FDA
Becky Schweighardt, Grace Science LLC
This session will address ways in which companies have tackled the CMC and regulatory challenges when bringing these life changing therapies to patients in need.
Session Speakers:
Manufacturing Challenges Limiting the Access to ATMPs
Ralf Altenburger, F. Hoffmann-La Roche Ltd.
Development of a Single Patient CRISPR Therapeutic
Richard Horgan, Cure Rare Disease
Additional Panelists:
Emmanuel Adu-Gyamfi, CBER, FDA
Becky Schweighardt, Grace Science LLC
Session Chairs
General
Tracks
Related Products
Plenary Session 5: Challenges with Incoming Materials - Raw, Starting, Ancillary, etc.
Raw material selection decisions early in the development of ATMPs are crucial and can impact the final product quality and ultimately patient safety…
Parallel Session 8: Approaches to Decentralized Manufacturing
Decentralized manufacturing involves the use of manufacturing units that can be deployed to multiple locations and in cases, to sites close to the patients…
Parallel Session 6: Allogeneic Cell-based Therapies: Next Frontier in Advanced Therapies
Allogeneic cell therapies have a potential to revolutionize life-saving Cell and Gene therapy (CGT) field by removing the bottlenecks created by long scheduling delays, lengthy manufacturing times and potential product failures of autologous cell therapies…
Keynote Presentation
Gene Therapy Drug Development for Ultra-Rare Disease: Challenges & Opportunities
