The newest gene therapy treatment, Casgevy, treats sick cell disease and transfusion-dependent beta thalassemia. Its addition means that CHLA provides the most cell and gene therapy treatments for pediatrics on the West Coast, according to a Jan. 27 system news release.
CHLA’s other cell and gene therapy treatments are:
- Elevidys: treats Duchenne muscular dystrophy
- Hemgenix: treats hemophilia B
- Kymriah: treats B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma
- Luxturna: treats inherited retinal disease
- Lyfgenia: treats sickle cell disease
- Omisirge: treats patients with blood cancers undergoing umbilical cord transplantation to minimize infection risks
- Roctavian: treats hemophilia A
- Zolgensma: treats spinal muscular atrophy
- Zynteglo: treats transfusion-dependent beta thalassemia
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