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

Executive Summary

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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Gene/Cell Therapies: Sponsors Must Plan For Long-Term Safety Follow-Up Even If They Fold

In final guidance documents on development of CAR-T cell products and human genome therapies incorporating genome editing, US FDA says that sponsors should plan for continued follow-up, with funding, even if they cease operations or withdraw the IND.

Gene Therapy: US FDA Labeling For Vertex’s Casgevy, Bluebird’s Lyfgenia Reflect Different Risks

Agency grants same-day approval to the first two gene therapies for sickle cell disease; bluebird’s lentiviral-based lovo-cel carries a boxed label warning on hematologic malignancies, while Vertex’s CRISPR-Cas9 exa-cel carries a warning and precaution about potential off-target effects. Only the Vertex product qualified for a rare pediatric disease priority review voucher.

Vertex’s Exa-Cel Off-Target Analyses Set Early Bar For Other Genome Editing Products

US FDA draft guidance does not specify the number of donor samples to test in nonclinical studies or the process for detecting genetic variants potentially susceptible to off-target effects. An advisory committee found adequate Vertex's analyses, which the sponsor called ‘the most comprehensive evaluation of off-target potential performed to date.’

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