FDA APPROVES A CRISPRCBASED MEDICINE FOR TREATMENT OF SICKLE CELL DISEASE

The Food and Drug Administration on Friday approved the world’s

rst 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 scientijc triumph for the technology that can e-ciently 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 a icts approximately 100,000 people in the U.S., a vast majority of whom are Black. The therapy, whose scientijc name is exa-cel, is described as a potential cure because the genetic Jx enabled by CRISPR is designed to last a lifetime, although conjrmation will require years of follow-up.

The FDA decision comes three weeks after regulators in the U.K. were the Jrst 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 cuttingedge therapies that provide potentially curative benejts.

“We are celebrating,” said Lewis Hsu, chief medical ofJcer at the Sickle Cell Disease Association of America and director of the pediatric sickle cell program at the University of Illinois at Chicago. “This decision has been a long time coming, pretty much since the Jrst description of sickle cell as a genetic disease some 70 years ago.”

Scientists Emmanuelle Charpentier and Jennifer Doudna published their Jrst CRISPR paper just over a decade ago. In 2020, the research won the pair a Nobel Prize. Re'ecting 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.