"FDA Approves CRISPR Gene-Editing Therapy for Beta-Thalassemia and Sickle Cell Disease"
TL;DR Summary
The FDA has approved Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy, a gene editing therapy, to treat beta-thalassemia, marking the first-ever CRISPR-edited therapy for the rare genetic blood disorder. Priced at $2.2 million for the one-time ex vivo treatment, the approval is for patients 12 years and older who require regular blood transfusions to survive, and it will compete with a gene-edited therapy from startup bluebird bio.
Topics:business#beta-thalassemia#crispr-therapeutics#fda#gene-editing-therapy#healthcare#vertex-pharmaceuticals
- FDA approves Vertex and CRISPR's Casgevy as first CRISPR-edited therapy for beta-thalassemia Endpoints News
- Vertex, Crispr Get Second FDA Approval for Gene Editing Therapy Bloomberg
- FDA expands approval of CRISPR-based medicine to treat beta thalassemia STAT
- CRSP Stock Dips Despite Snagging A Second Gene-Editing OK; VRTX Stock Continues On Its Warpath Investor's Business Daily
- New trial at MUSC could present low-risk treatment option for sickle cell disease Live 5 News WCSC
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