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Vertex’s Exa-Cel: Off-Target Gene Editing Analyses Sufficient Given Robust Clinical Efficacy, FDA Panel Says

 
• By Sue Sutter

Weighing Vertex’s evaluation of theoretical risks of off-target editing with the gene therapy’s strong clinical results in sickle cell disease, advisory committee says in silico and cellular analyses were sufficient but suggests closer monitoring of potential on- and off-target effects postapproval.  

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Theoretical analyses of off-target effects may eventually lead to diminishing returns, an FDA panelist said. • Source: Shutterstock

Vertex Pharmaceuticals Incorporated’s nonclinical analyses for potential off-target effects of its sickle cell disease gene therapy exa-cel are sufficient, particularly given the robust clinical efficacy seen in a disease with high unmet need, a US Food and Drug Administration advisory committee said on 31 October.

However, the Cellular, Tissue and Gene Therapies Advisory Committee said long-term follow-up of patients receiving the treatment, which is made using CRISPR/Cas9 gene editing technology, should include whole genome sequencing and monitoring to track on-target

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