On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved two innovational gene therapies, Casgevy and Lyfgenia, for treating sickle cell disease (SCD) in patients aged 12 and older. This marks the first approval of cell-based gene therapies for SCD, with Casgevy also being the first to use CRISPR/Cas9 genome editing technology.

SCD, primarily affecting African and Hispanic Americans, involves a mutation in hemoglobin that leads to red blood cells developing a sickle shape. This mutation causes severe pain, organ damage, and potentially life-threatening complications.

Casgevy is designed for patients with recurrent vaso-occlusive crises (VOCs), which employs CRISPR/Cas9 to edit patients’ hematopoietic stem cells. These modified cells, once reinfused into the patient, increase fetal hemoglobin production, reducing red blood cell sickling. Lyfgenia, intended for patients with a history of vaso-occlusive events (VOEs), uses a lentiviral vector to modify stem cells to produce HbAT87Q, a therapeutic hemoglobin that lowers the sickling risk through cell-based gene therapy. Both treatments involve collecting and modifying the patient’s own blood stem cells, followed by a one-time infusion after high-dose chemotherapy (myeloablative conditioning).

Clinical trials demonstrated Casgevy’s efficacy, with over 93% of patients achieving freedom from severe VOCs. Lyfgenia showed 88% of patients had complete resolution of VOEs. Common side effects for both include low blood cell counts and mouth sores. Lyfgenia carries a black box warning for the risk of blood cancer.

These therapies, which received Priority Review and other FDA designations, are developed by Vertex Pharmaceuticals Inc. (Casgevy) and Bluebird Bio Inc. (Lyfgenia). The FDA’s approval highlights its commitment to advancing treatments for severe health conditions.

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