FDA Approves Gene Therapy for Inherited Blood Disorder

The treatment for Beta-Thalassemia is expected to cost $2.2 million and will be available in the United States this year.

Staff members at Oregon Health & Science University's Casey Eye Institute perform the first-ever in vivo CRISPR gene edit procedure on March 21, 2020. OHSU/Kristyna Wentz-Graff

The U.S. Food and Drug Administration (FDA) has approved a gene therapy to treat an inherited blood disorder, even as the agency earlier expressed concerns about the technology leading to “unintended” gene edits.

“Casgevy, a cell-based gene therapy,“ was approved ”for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassemia, an inherited disorder characterized by life-long anemia requiring frequent blood transfusions,” the FDA said in a Jan. 16 announcement.