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Gene Editing: Exa-Cel Panel Review Raises Practical Concerns About Theoretical Risks

 
• By Sue Sutter

US FDA advisory committee members questioned how potential off-target effects with Vertex’s gene therapy for sickle cell disease should be conveyed to patients and families, and whether labeling should say anything about these theoretical risks.

Mountain terrain
The exa-cel adcomm found itself navigating new terrain of practical concerns befitting the cutting-edge technology involved. • Source: Shutterstock

The first US Food and Drug Administration advisory committee called upon to review a therapeutic incorporating genome editing found itself navigating new terrain befitting this cutting-edge technology, including practical implications of theoretical risks.

Specifically, panelists questioned how potential off-target effects with Vertex Pharmaceuticals Incorporated’s exa-cel should be conveyed to patients and their...

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Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
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