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FDA Approves Genetic Treatments for Sickle Cell Disease

PORTLAND — The Sickle Cell Anemia Foundation of Oregon is excited by the news that two types of gene therapy for sickle cell have been approved by the FDA: Casgevy for “gene editing”, and Lyfgenia for “gene addition”.

The SCDAA has called gene therapy “potentially curative“, though, they warn, not everyone may be “eligible for gene therapy.”

Gene therapy involves a complex series of steps, including sampling and treating the patient’s stem cells, a regime of chemotherapy where the abnormal cells are removed, and an infusion of the patient’s own cells treated for sickle cell disease.

Patients who have undergone such treatments can expect acute episodes of the disease to diminish significantly over time as diseased blood cells are naturally replaced by healthy cells.

Candidates for sickle cell gene therapy must be evaluated against the dangerous effects of chemotherapy.

The FDA has approved both Casgevy and Lyfgenia for sickle cell patients ages 12 and up. The treatments are approved for sickle cell phenotypes SS and S-beta-zero.

SCDAA has more information here.