FDA approves cure for sickle cell disease, the first treatment to use gene-editing tool CRISPR

Gene-editing treatment for sickle cell approved by FDA

(WJW) – Breakthrough treatments for sickle cell disease have been approved by the U.S. Food and Drug Administration (FDA). On Dec. 8, the FDA approved Casgevy and Lyfgenia. They’re the first cell-based gene therapies for the treatment of sickle cell patients 12 and older. See the research here Casgevy is also the first FDA-approved treatment to use a novel genome editing technology. Sickle cell affects about 100,000 people in the U.S. Si…

FDA approves new sickle cell disease treatment

Dr. Jeff Pothoff with University of Wisconsin Health explains sickle cell treatment which uses gene-editing tool CRISPR to prevent red blood cell 'sickling'

FDA Approves Sickle Cell Disease Gene Therapy

The FDA announced Friday that it had approved two gene therapies based on CRISPR technology for sickle cell disease, the first cell-based treatments for the disease. The gene-based therapies, Casgevy and Lyfgenia, are for use in patients 12 years old and older. Both of these therapies are produced by using a patient’s own stem cells. Casgevy is manufactured by Vertex Pharmaceuticals Inc., while Lyfgenia is manufactured by Bluebird Bio Inc. “Sick…

‘Remarkable’ gene-editing treatment for sickle cell disease is approved by FDA

Modern medicine is one step closer to potentially curing sickle cell disease. On the heels of the U.K.'s authorizing the world’s first gene-edited treatment for sickle cell disease on Nov. 16, the U.S. Food and Drug Administration (FDA) approved the same treatment on Dec. 8. "CASGEVY’s approval by the FDA is momentous," Reshma Kewalramani, M.D., chief executive officer and president of Vertex Pharmaceuticals in Boston, said in a statement provid…

US approves first gene-editing treatment for sickle cell patients

The United States Food and Drug Administration has approved a gene-editing treatment for sickle cell patients from 12 years of age and above. According to a press statement released by the FDA on Saturday, the treatment comes in two stages namely Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle

The American authorities have approved: The first gene therapy using the CRISPR gene scissors may be used in the USA. A milestone, say experts. And that could just be the beginning. By V. Simon. [more]