FDA Approves Gene Editing Treatment for Sickle Cell Disease
F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR
On Friday, the Food and Drug Administration approved the first gene editing therapy ever to be used in humans, for sickle cell disease, a debilitating blood disorder caused by a single mutated gene.
The agency also approved a second treatment using conventional gene therapy for sickle cell that does not use gene editing.
For the 100,000 Americans with the disease, most of them Black, the approvals offer hope for finally living without an affliction that causes excruciating pain, organ damage and strokes.
US FDA approves two gene therapies for sickle cell disease
The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.
Casgevy, developed by partners Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN), and bluebird bio's (BLUE.O) Lyfgenia were approved for people aged 12 years and older.
Two new cutting-edge sickle cell treatments approved by FDA despite side effects
For the first time in history, there’s hope that the debilitating illness known as sickle cell disease may be treatable — even cured.
The Food and Drug Administration announced Friday that two gene therapies for sickle cell disease have been approved following years of clinical trials.
Sickle cell disease is an inherited blood disorder caused by a mutated gene that affects hemoglobin, a protein in red blood cells, according to Johns Hopkins Medicine.