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healthcare2 years ago

"FDA Grants Dual Approval for Vertex/CRISPR Gene Therapy in Treating Inherited Blood Disorders"

The US FDA has approved Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy, Casgevy, for treating transfusion-dependent beta thalassemia in patients 12 years and older, making it the second US approval for the therapy. Casgevy, priced at $2.2 million, is the first treatment based on CRISPR gene editing technology to secure approval for this disorder in the United States, with an estimated peak sales of about $400 million. This therapy offers hope for patients with the severe form of the disorder, which causes life-threatening anemia requiring frequent blood transfusions.