Two gene therapies for sickle-cell disease (SCD) are:
1. Lyfgenia from Bluebird Bio
2. Casgevy by Vertex Pharmaceuticals and Crispr Therapeutics
This transformative therapy depend on Crispr gene-editing technology and uses the patients' own blood stem cells. The DNA is cut at targeted areas, and the faulty DNA deleted, added or replaced. The modified blood stem cells are transplanted back into the patient where they multiply within the bone marrow.
The gene therapy is a one-time treatment.
This therapy is approved for patients aged 12 and older.
