Becker's Healthcare December 8, 2023
On Dec. 8, the FDA approved the nation’s first medicine based on CRISPR gene-editing technology to be used as a treatment for patients with sickle cell disease, a major scientific advancement that opens the door for future gene therapy approvals for inherited disorders.
The FDA approved exagamlogene autoemcel, or exa-cel, marketed as Casgevy, to treat sickle cell disease in patients 12 and older. The medicine is made by Vertex Pharmaceuticals and CRISPR Therapeutics. It’s meant as a one-time therapy that works by using CRISPR technology to remove the disease-causing gene from a patient’s stem cells.
“As scientists, the fact that we have arrived at a potential curative treatment for sickle cell disease in a relatively brief period is...
Today's Sponsors
Today's Sponsor