Crispr Is Ready for Its Commercial Debut
The gene-editing treatment has been transformative for patients with sickle cell disease. It’s time for the FDA to move it past clinical trials.
Set to get a green light.
Photographer: Scott Eisen/Bloomberg
The power and peril of Crispr was up for debate at a Food and Drug Administration advisory committee meeting yesterday. Their task: to consider the risks and benefits of a gene therapy for sickle cell disease developed by Vertex Pharmaceuticals Inc. and Crispr Therapeutics Inc. Given the positive tenor of the meeting, the therapy is expected in early December to become the first Crispr-based therapy to reach the market.
The focus of the day wasn’t whether the treatment works. FDA’s advisers agreed that it is undeniably transformative for most of the people who have received it so far. Only one of the 30 patients followed for at least 18 months has since experienced the disease’s excruciating vaso-occlusive crises, which occur when misshapen blood cells — the hallmark of the genetic disease — get stuck and block blood flow. During the meeting, several patients offered moving testimony about the profound effect the treatment has had on their life, describing freedom from pain, the ability to work, and enjoying simple pleasures with their families.