U.S. Agency Authorizes Millions Of Dollars' Worth Of Gene-editing Therapies For Sickle Cell Disease

The FDA approved the first gene therapies for sickle cell anemia

The two approved treatments, Casgevy and Lyfgenia, will cost $2.2 million and $3.1 million

A treatment for sickle cell disease has received FDA approval in the United States.

The treatment, known as Casgevy, is the first gene-editing tool approved in the United States and was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. Its creators were awarded the 2020 Nobel Prize in Chemistry.

The approval represents the first of two possible developments in the treatment of the hereditary blood disorder. Lyfgenia, a gene therapy developed by pharmaceutical company Bluebird Bio, was given FDA approval on Friday as an additional sickle cell disease treatment. The genetic alteration of a patient's own stem cells is how both therapies function.

Up until recently, the sole recognized treatment for sickle cell disease involved receiving a bone marrow transplant from a donor; however, this procedure entailed the challenge of finding a matching donor and the possibility of immune system rejection.

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Casgevy eliminates the need for a donor and was approved for use in patients 12 years of age and up. It modifies the DNA in a patient's stem cells using CRISPR to eliminate the disease-causing gene. The new treatment costs $2.2 million per patient, according to NBC News.

Before the experts declare this a cure, patients must be monitored for an extended period of time. Although gene editing may not be suitable for every sickle cell disease patient, it is anticipated to cost several million dollars per patient. Furthermore, it wouldn't stop the gene from being inherited by subsequent generations.