VRTX - Gene Therapy For Beta-Thalassemia: CRISPR Therapeutics/Vertex's Casgevy Secures FDA Nod Ahead of Schedule | Benzinga
Tuesday, the FDA approved CRISPR Therapeutics AG’s (NASDAQ:CRSP)/Vertex Pharmaceutical Incorporated’s (NASDAQ:VRTX) Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older.
The approval comes two months before the PDUFA date of March 30, 2024.
Vertex is engaging with experienced hospitals to establish a network of independently operated treatment centers (ATCs) throughout the U.S. to offer Casgevy to patients.
Also Read: CRISPR Therapeutics’ Gene-Edited Oncology Pipeline To Focus On Expanded Use In Autoimmune Diseases.
Casgevy is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy ...