Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, the treatment, called Casgevy, involves collecting stem cells from a patient’s blood, which are then genetically modified using CRISPR technology and injected back into the patient. In addition to this therapy, the FDA has also approved another gene therapy to treat the same disease.
Sickle cell anemia is a relatively rare but serious genetic disease that disproportionately affects black people. It is associated with pain and the need for regular blood transfusions.
So far, the only treatment is bone marrow transplantation from a donor, which is associated with a risk of transplant rejection. The new treatment — used once but requiring a hospital stay of several weeks — currently costs $2.2 million. from the patient.
CRISPR is a new and revolutionary gene editing method, discovered in 2012 by Jennifer Doudna and Emmanuel Charpentier, for which they received the 2020 Nobel Prize in Chemistry. The FDA’s decision to approve a drug based on this method could pave the way for more treatments of this type.
As Doudna said Friday, the drug’s approval “builds on the promise of gene therapy that seeks to treat disease at its source through targeted changes in a person’s DNA.”
The Nobel laureate was quoted by the Wall Street Journal as saying: “It almost changes the way we define medicine.
The development was also praised by US President Joe Biden, who described the treatment as the result of government investments in innovation.
“This significant medical advance holds great promise for the development of additional life-saving therapies and gives hope to the millions of Americans living with other rare diseases,” Biden said in a statement.
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