The US Food and Drug Administration (FDA) achieved a milestone on Friday by approving a groundbreaking gene-editing therapy designed to address sickle cell disease (SCD), marking the inaugural approval of CRISPR gene editing by federal regulators.
The FDA authorized two novel treatments for SCD: Casgevy and Lyfgenia, The Hill reported.
Casgevy is the outcome of a collaboration between Vertex Pharmaceuticals and CRISPR Therapeutics. The innovative treatment involves utilizing the patient's own stem cells, which are then genetically edited to enhance fetal hemoglobin production before being reintroduced into the individual.
Lyfgenia is another cell-based gene therapy that genetically modifies stem cells to create HbAT87Q, a hemoglobin that prevents sickle-shaped red blood cells.
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Sickle cell disease is an inherited blood disorder characterized by misshapen half-moon red blood cells, causing blockages in blood vessels and triggering excruciating crises.
Casgevy leverages CRISPR technology to edit a person's DNA, activating fetal hemoglobin, a protein that typically declines after birth. In clinical trials, Casgevy demonstrated efficacy by eliminating pain crises in the majority of patients.
