In both therapies, stem cells are removed from a patient’s blood for treatment<\/p>\n<\/h3>\n
By Physician\u2019s Briefing Staff HealthDay Reporter<\/b><\/p>\n
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FRIDAY, Dec. 8, 2023 (HealthDay News) — Two milestone gene therapies for sickle cell disease have been approved by the U.S. Food and Drug Administration.<\/p>\n
Casgevy is the first medicine available in the United States to treat a genetic disease using the CRISPR gene-editing technique. The one-time treatment permanently changes DNA in a patient’s blood cells, freeing them from the excruciating symptoms of sickle cell disease for life. The other approved therapy, Lyfgenia, uses a common virus to deliver genetic modifications to a patient’s blood stem cells in their bone marrow.<\/p>\n
In both therapies, stem cells are removed from a patient’s blood for treatment. With Casgevy, CRISPR gene-editing technology knocks out a gene that triggers the development of defective, crescent-shaped blood cells. Meanwhile, medicine kills off flawed blood-producing cells in patients, who are then given back their own altered stem cells. The same thing happens with Lyfgenia, only a virus is used to deliver a genetic payload that causes the blood cells to start producing healthy hemoglobin. The genetically modified blood stem cells are then given back to the patient as a one-time, single-dose infusion.<\/p>\n
Evidence supporting the approval of Casgevy was from a study of 44 patients treated with the gene-editing process. Out of 31 patients with sufficient follow-up time to be evaluated, 29 had complete freedom from the severe pain and organ damage that can occur during a sickle cell exacerbation. Furthermore, no patients experienced graft failure or graft rejection. Approval of Lyfgenia was supported by a 24-month study in which 32 patients received the therapy. Of those, 28 had no sickle cell events following their transplant.<\/p>\n
Approval of Casgevy and Lyfgenia was granted to Vertex Pharmaceuticals and Bluebird Bio, respectively.<\/p>\n