(NEWSnet/AP) — Regulators on Friday approved two new gene types of gene therapy for sickle cell disease that doctors hope can cure the inherited blood disorder that afflicts mostly Black people.
U.S. Food and Drug Administration said the one-time treatments can be used for patients age 12 and up with severe forms of the disease. One, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first approved therapy based on CRISPR, a gene-editing tool. The other is manufactured by Bluebird Bio and works differently.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need,” FDA’s Dr. Nicole Verdun said. “"We are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease.”
In the U.S., an estimated 100,000 people have the disease and about 20% of them have the severe form.
Sickle cell is most common among Black people. About one in 365 are born with the disease U.S.. Scientists believe being a carrier of the sickle cell trait helps to protect against severe malaria, so the disease occurs more often in mosquito-prone regions such as Africa or in people whose ancestors lived in those places
The disease affects hemoglobin, the protein in red blood cells that carries oxygen. A genetic mutation causes the cells to become sickle or crescent-shaped, which can block blood flow, causing excruciating pain, organ damage, stroke and other problems.
Current treatments include medication and blood transfusion. The only permanent solution is a bone marrow transplant, which must come from a closely matched donor without the disease and brings a risk of rejection.
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