FDA Reviewers Question Safety of Sickle Cell Gene Therapy

October 30, 2023

(MedPage Today) – Whether the investigational sickle cell disease therapy exagamglogene autotemcel (exa-cel) has adequately proven that it won’t produce off-target genetic changes was questioned by FDA reviewers ahead of an advisory committee meeting. The gene-editing CRISPR technology underpinning exa-cel — a product of a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics — is the first of its kind to come before the agency for an approval decision. Exa-cel has demonstrated positive results in patients with transfusion-dependent beta-thalassemia and severe sickle cell diseaseopens in a new tab or window. (Read More)