La Semana



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.

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