A Sickle Cell Breakthrough Is Here. Now the Hard Part.
The first Crispr treatment to make it to the commercial market is going to open up new possibilities — and with them, new challenges.
Patients with sickle cell disease have waited a long time for effective treatments.
Photographer: Erin Clark/The Boston Globe/Getty ImagesThe approval of Casgevy, Vertex Pharmaceuticals Inc. and Crispr Therapeutics Inc.’s gene therapy for sickle cell disease, is a transformative moment in medicine. Not only is it the first Crispr-based drug to reach the market — it’s a potentially life-altering advance for a patient population that has been for too long ignored and underserved by the medical establishment. Lyfgenia, a second gene therapy from Bluebird Bio that uses different technology, was also given the Food and Drug Administration’s green light.
Now comes the hard work of treating patients.