ARTICLE | Regulation
Exa-cel meeting clarifies off-target expectations for gene editing therapies
FDA’s advisers were satisfied with the methods used by CRISPR Therapeutics and Vertex to analyze the off-target edits of gene editing therapy exa-cel
November 1, 2023 11:18 PM UTC
The theoretical risk of off-target edits can not be ruled out for exa-cel — the first gene editing therapy up for FDA approval — but that doesn’t mean the treatment should be withheld from patients.
Rather than showstoppers, off-target edits are just one more risk to factor into the risk-benefit analyses of new CRISPR-based therapeutic products, according to FDA’s Cellular, Tissue and Gene Therapies Advisory Committee, which met on Tuesday to discuss off-target edits associated with exagamglogene autotemcel (exa-cel) from CRISPR Therapeutics AG (NASDAQ:CRSP) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX). ...