Sickle cell treatment created with gene editing gains UK approval
The first CRISPR-based treatment is expected to receive US approval next month. But it could cost millions of dollars per patient.
British regulators on Thursday approved the first treatment derived from CRISPR, the revolutionary method of gene editing. Called Casgevy, the treatment is intended to cure sickle cell disease and a related disease, beta thalassemia.
The makers, Boston-based Vertex Pharmaceuticals and CRISPR Therapeutics, About 2,000 British patients with sickle cell disease or beta thalassemia are estimated to be eligible for its treatment.
The companies expect the Food and Drug Administration to will approve Casgevy for sickle cell patients in the United States in early December. The agency will decide whether to approve beta thalassemia next year.
In late December, the F.D.A. is expected to approve another sickle cell gene therapy by Bluebird Bio of Somerville, Massachusetts. This treatment does not rely on gene editing, but rather a method that inserts new DNA into the genome.
Sickle cell disease is caused by a defective gene that results in the creation of abnormal hemoglobin, the component that carries oxygen in red blood cells. The cells themselves become malformed, causing episodes of extreme pain. About 100,000 Americans, mostly black and Hispanic, are thought to have the disease.
In beta thalassemia, the defective gene causes a high rate of Deficient hemoglobin in red blood cells. . The condition is rare.
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The first CRISPR-based treatment is expected to receive US approval next month. But it could cost millions of dollars per patient.
British regulators on Thursday approved the first treatment derived from CRISPR, the revolutionary method of gene editing. Called Casgevy, the treatment is intended to cure sickle cell disease and a related disease, beta thalassemia.
The makers, Boston-based Vertex Pharmaceuticals and CRISPR Therapeutics, About 2,000 British patients with sickle cell disease or beta thalassemia are estimated to be eligible for its treatment.
The companies expect the Food and Drug Administration to will approve Casgevy for sickle cell patients in the United States in early December. The agency will decide whether to approve beta thalassemia next year.
In late December, the F.D.A. is expected to approve another sickle cell gene therapy by Bluebird Bio of Somerville, Massachusetts. This treatment does not rely on gene editing, but rather a method that inserts new DNA into the genome.
Sickle cell disease is caused by a defective gene that results in the creation of abnormal hemoglobin, the component that carries oxygen in red blood cells. The cells themselves become malformed, causing episodes of extreme pain. About 100,000 Americans, mostly black and Hispanic, are thought to have the disease.
In beta thalassemia, the defective gene causes a high rate of Deficient hemoglobin in red blood cells. . The condition is rare.
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