"FDA Grants Dual Approval for Vertex/CRISPR Gene Therapy in Treating Inherited Blood Disorders"
TL;DR Summary
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.
Topics:business#crispr-therapeutics#gene-therapy#healthcare#transfusion-dependent-beta-thalassemia#us-fda#vertex-pharmaceuticals
- US FDA approves Vertex/CRISPR gene therapy for an inherited blood disorder CNBC
- FDA clears first CRISPR treatment for a second disease, beta thalassemia CNN
- CRSP Stock Dips Despite Snagging A Second Gene-Editing OK; VRTX Stock Continues On Its Warpath Investor's Business Daily
- Vertex, Crispr Get Second FDA Approval for Gene Therapy Bloomberg
- CRISPR-based therapy receives expanded approval for beta thalassemia STAT
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