Health
Vertex, Crispr Get Second FDA Approval for Gene Therapy
- New treatment for beta thalassemia to compete with Bluebird
- FDA approved Casgevy to treat sickle cell disease in December
This article is for subscribers only.
The US Food and Drug Administration has approved the first Crispr-based treatment for a second condition, clearing the gene editing therapy for the blood disorder beta thalassemia.
The drug, called Casgevy, is a collaboration between Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG. It was expected to be approved in March, but was cleared early. Casgevy will compete with Zynteglo, a gene therapy for beta thalassemia sold by the biotech company Bluebird Bio Inc.