The U.S. Food and Drug Administration approved Casgevy, a $2.2 million per patient treatment for use in patients with sickle cell disease.
Casgevy, which utilizes CRISPR technology to edit a person’s genes, was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
BREAKING NEWS $CRSP
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease https://t.co/lxb2diXT9r
— Gene Investing w/Anthony 🧬 (@GeneInvesting) December 8, 2023
The approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement. Yet reaching the tens of thousands of people who could benefit from the treatment could be challenging given the potential hurdles — including cost — of administering the complex therapy.
Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit a person’s genes to treat disease. The treatment was approved by U.K. regulators last month.
Sickle cell, an inherited blood disorder, causes red blood cells to become misshapen half moons that get stuck inside blood vessels, restricting…
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