Forbes January 16, 2024
James Farrell

Topline

The Food and Drug Administration approved the use of Casgevy, a therapy from Vertex Pharmaceuticals and CRISPR Therapeutics that uses CRISPR gene-editing to treat the serious blood disorder transfusion-dependent beta thalassemia—marking the second major U.S. regulatory approval for the emerging gene-editing technology.

Key Facts

What To Watch For

Vertex says it’s engaging with experienced hospitals to establish “a network of independently operated, authorized treatment centers” throughout the U.S. to administer Casgevy. There are currently nine activated treatment centers in the U.S., but more will be activated in the coming weeks, Vertex said in a press release. The administration of Casgevy requires experience in stem cell transplantation.

Key Background

CRISPR, or “clustered regularly interspaced short palindromic repeats,” is seen as...

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