US FDA approves Vertex/CRISPR gene therapy for an inherited blood disorder (updated)

Crispr Therapeutics AG

16/01/2024 20:02

(Adds details in paragraphs 2 & 4)
       Jan 16 (Reuters) - The U.S. Food and Drug Administration
(FDA) has approved Vertex Pharmaceuticals  VRTX.O  and CRISPR
Therapeutics'  CRSP.BN  gene therapy to treat a rare blood
disorder requiring regular blood transfusions, in patients 12
years and older, Vertex said on Tuesday.
    The therapy, branded as Casgevy, was approved for
transfusion-dependent beta thalassemia and is based on the Nobel
Prize-winning CRISPR gene editing technology.
    The decision earns Casgevy the second approval in the United
States after the health regulator, in December, greenlighted it
for sickle cell disease, another inherited blood disorder.
        Casgevy requires administration through authorized
treatment centers with experience in stem cell transplantation,
Vertex said.

 (Reporting by Mariam Sunny in Bengaluru; Editing by Shailesh
Kuber)
 ((Mariam.ESunny@thomsonreuters.com;))