FDA approves first gene editing therapies for sickle cell disease

The U.S. Food and Drug Administration (FDA) has approved two groundbreaking cell-based gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease (SCD) in patients aged 12 and older. Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first FDA-approved treatment using CRISPR/Cas9 genome editing technology. The therapy modifies patients’ hematopoietic (blood) stem cells using CRISPR/Cas9 technology to turn on fetal hemoglobin, preventing the sickling of red blood cells. Lyfgenia, developed by Bluebird Bio, uses a lentiviral vector for genetic modification, producing HbAT87Q to reduce the risk of sickling. Both therapies involve a one-time, single-dose infusion as part of a hematopoietic stem cell transplant. The FDA granted Priority Review, Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy designations to both treatments.

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