Blood disorder that can lead to premature death affects estimated 100,000 people in the United States, most of whom are Black
The US Food and Drug Administration has approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene-editing technology, opening up two “transformative therapy” avenues for some patients.
The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and Crispr Therapeutics. Both therapies are made from the patients’ own blood stem cells and were approved for people aged 12 and older.
The Vertex/Crispr gene therapy uses the breakthrough gene-editing technology that won its inventors the Nobel prize in 2020. The therapy can be directed to cut DNA in targeted areas, enabling the ability to accurately remove, add or replace DNA where it was cut.
The modified blood stem cells are then transplanted back into the patient, where they attach and multiply within the bone marrow and increase the production of fetal hemoglobin, a type of hemoglobin that facilitates oxygen delivery.
Lyfgenia is a cell-based gene therapy that modifies a patient’s blood stem cells to produce a gene therapy-derived hemoglobin that functions similarly to a type of normal adult hemoglobin not affected by sickle cell disease.
