CRISPR/Vertex gene editing therapy approved in U.S. for beta thalassemia

ipopba Vertex Pharmaceuticals ( NASDAQ: VRTX ) announced Tuesday that the U.S. FDA approved its groundbreaking gene editing therapy, Casgevy, developed with CRISPR Therapeutics ( NASDAQ: CRSP ) for those aged 12 years and older with transfusion dependent beta thalassemia (TDT). The approval comes weeks after the agency cleared Casgevy for sickle cell disease, making it the first U.S.

approved gene editing therapy. At the time, Casgevy, a CRISPR/Cas9 gene edited cell therapy, was under FDA review for TDT with a target action date of March 30. "On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date," Reshma Kewalramani, CEO of Vertex ( VRTX ), remarked.

The company also said that nine clinical centers in the U.S. are now authorized to offer Casgevy for TDT patients, and additional sites will be launched in the coming weeks. According to Boston Children's Hospital, there are at least 1,200 patients with TDT in the US. More on CRISPR, Vertex Pharma, etc.

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