Health
Vertex, Bluebird Both Get FDA Approval for Sickle Cell Gene Therapies
- Decision is a milestone for the Nobel-winning Crispr discovery
- FDA also approves Bluebird’s therapy with cancer risk warning
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The US Food and Drug Administration approved the first gene editing therapy using Crispr technology on Friday, a collaboration between Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG for treating sickle cell disease.
Called Casgevy, the approval will allow the drug to come to market in the US. Sickle cell disease affects some 100,000 Americans. Vertex says Casgevy is meant for the around 20,000 that have a severe version of the illness. The disease can land some people in the hospital multiple times a year.