The United States Food and Drug Administration (FDA) has approved a treatment for sickle cell disease.
The therapy, called Casgevy,
from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to
be approved in the United States that uses the gene-editing tool CRISPR, which
won its inventors the Nobel Prize in Chemistry in 2020.
The approval marks the first of
two potential breakthroughs for the inherited blood disorder.
The FDA on Friday also approved
a second treatment for sickle cell disease, called Lyfgenia, a gene therapy
from drugmaker Bluebird Bio. Both treatments work by genetically modifying a
patient’s own stem cells.
Until now, the only known cure
for sickle cell disease was a bone marrow transplant from a donor, which
carries the risk of rejection by the immune system, in addition to the
difficult process of finding a matching donor.
Casgevy, which was approved for
people ages 12 and older, removes the need for a donor. Using CRISPR, it edits
the DNA found in a patient’s stem cells to remove the gene that causes the
disease.
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