Error loading player: No playable sources found

89833

Plenary Session 4 - Potency Assays for Gene Edited Products

Date
June 12, 2024
This product is not available for individual purchase, but it is available as part of the following products:

Measurement of the biological activity is crucial for lot release, stability and any comparability exercise of all biological medicinal products. Potency assays should be implemented as early in clinical developments as possible because correlation with clinical performance could help in future identification of sub potent batches. However, development of an appropriate potency assay is often challenging, especially for products with complex or not well-defined mechanisms of action.  

Gene editing (GE) tools are used to achieve targeted modifications of genome sequences resulting in gene inactivation, modifications or insertions at specific locations, either in vivo or ex vivo. This allows the generation of very diverse cell phenotypes that can be used in the clinic to treat a high array of diseases. Thus, developing specific potency assays for gene edited medicinal products can have additional complications.  


This session will aim to review FDA’s recently released draft guideline on potency assurance for cell and gene therapy products. It will also cover issues to be considered when designing a potency assay for ex vivo genome-edited products or products that are intended to edit the genome in vivo, considering the different GE tools and possible mechanisms of actions. The session will also present the particular challenges of developing a potency assay for a GE product intended to knock-out a gene.  


Session Speakers:


Potency Assurance for Cellular and Gene Therapy Products

Andrew Byrnes, CBER, FDA


Potency Development for an in Vivo AAV Gene Editing Therapy

Debaditya BhattacharyaElevateBio


Approaches to Potency Assays for CRISPR Genome Editing Therapeutics 

Kristy WoodIntellia Therapeutics, Inc.


Additional Panelist:


Keith WonnacottLexeo Therapeutics, Inc.


Related Products

Thumbnail for Parallel Session 2 - How to Control Off Target Genotoxicity for Genome and Epigenome Editing Medicines
Parallel Session 2 - How to Control Off Target Genotoxicity for Genome and Epigenome Editing Medicines
Genome editing is a tool that enables a new generation of medicines by directly targeting with high precision the genetic cause of a disease. It also allows for immune evasion and prevents microenvironment responses: concepts vital to more potent, off the shelf, cell, and gene therapies…
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 Hot Topic Session - ICH Cell & Gene Therapy Discussion Group Updates
Hot Topic Session - ICH Cell & Gene Therapy Discussion Group Updates
The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) brings together regulatory authorities and representatives from the pharmaceutical industry from across the globe to discuss a range of technical and scientific considerations for medicinal…