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Gene Editing: US Panel Review Of Vertex’s Exa-Cel To Focus On Nonclinical Assessment Of Off-Target Effects

 
• By Sue Sutter

Advisory committee discussion on the adequacy of Vertex’s analyses, using in silico and cellular methods, of off-target alterations with the gene therapy for sickle cell disease could help guide nonclinical assessment of future products made with CRISPR/Cas9-mediated gene editing.

Missed target
Vertex's analyses of off-target effects for exa-cel is the focus of an adcomm review. • Source: Shutterstock

A US Food and Drug Administration advisory committee review of Vertex Pharmaceuticals Incorporated’s exa-cell gene therapy for sickle cell disease will help inform how potential off-target effects should be assessed for future products produced by genome editing.

On 31 October, the Cellular, Tissue and Gene Therapies Advisory Committee will discuss Vertex’s exagamglogene autotemcel (exa-cel) for treatment of sickle cell disease (SCD) in

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