World-first gene therapy for 2 blood disorders — sickle cell and thalassemia — approved

Casgevy the 1st medicine licensed using the gene editing tool CRISPR

Excerpt from the Press Release:

Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the disease in the U.K. 

In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020.

The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over.

Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics. To date, bone marrow transplants, extremely arduous procedures that come with very unpleasant side effects, have been the only long-lasting treatment.

“The future of life-changing cures resides in CRISPR-based [gene-editing] technology,” said Dr. Helen O’Neill of University College London.

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Both sickle cell disease and thalassemia are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.

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