Fierce Biotech November 1, 2023
FDA advisers appear satisfied with CRISPR Therapeutics and Vertex Pharmaceuticals’ assessment of potential off-target effects from their CRISPR-based gene therapy exa-cel, quelling the top concern from the FDA as an approval deadline nears.
Acting chair of the Cellular, Tissue, and Gene Therapies Advisory Committee Taby Ahsan, Ph.D., summarized Tuesday’s meeting by noting that there was a “robust approach using multiple methods to try to identify these off-targets.”
The conclusion bolsters CRISPR Tx and Vertex’s stance that the potential—or lack thereof—for off-target edits has been studied in enough detail to warrant approval given the potential benefit for patients with sickle cell disease. The FDA is set to decide by December 8 on whether exa-cel will be approved for sickle cell...
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