FDA Approves CRISPR-Based Medicine For Treatment of Sickle Cell Disease

An anonymous reader quotes a report from STAT: The Food and Drug Administration on Friday approved the world's first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations -- ushering in a new era of genetic medicines for inherited diseases. In a clinical trial, Casgevy was shown to eliminate recurrent episodes of debilitating pain caused by sickle cell, which afflicts approximately 100,000 people in the U.S., a vast majority of whom are Black. The therapy, whose scientific name is exa-cel, is described as a potential cure because the genetic fix enabled by CRISPR is designed to last a lifetime, although confirmation will require years of follow-up. The FDA decision comes three weeks after regulators in the U.K. were the first to clear the drug. Approval in the European Union is expected next year. The FDA is also expected to rule on exa-cel as a treatment for beta thalassemia, another inherited blood disorder, by March 30. The FDA on Friday also approved another sickle cell treatment, a gene therapy from Bluebird Bio called Lyfgenia. Patients will now have the option of two cutting-edge therapies that provide potentially curative benefits. Scientists Emmanuelle Charpentier and Jennifer Doudna published their first CRISPR paper just over a decade ago. In 2020, the research won the pair a Nobel Prize. Reflecting on the approval of Casgevy, Charpentier told STAT via email that she was "excited and pleased" for what it means for patients and their families.

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