FDA approves two sickle cell disease gene therapies, one based on CRISPR
Dec. 30 (ZFJ) — The U.S. Food and Drug Administration approved two cell-based gene therapies, Casgevy and Lyfgenia, to treat sickle cell disease in patients 12 years and older, the agency announced on Dec. 8, 2023. Casgevy is the first FDA-approved treatment to use CRISPR/Cas9 gene-editing technology.
Sickle cell disease is a group of inherited blood disorders. It affects about 100,000 Americans, is most common in African Americans, and also affects Hispanic Americans.