Nine hospitals will provide a first-of-its-kind cell-based gene therapy for patients with sickle cell disease following the Food and Drug Administration's recent approval of the treatment.
The hospitals that will offer CRISPR Therapeutics' Casgevy genome-edited cell therapy are Boston Medical Center; Children’s National Hospital in Washington, D.C.; City of Hope Children’s Cancer Center in Los Angeles; Medical City Children’s Hospital in Dallas; Methodist Children’s Hospital in San Antonio, Texas; Nationwide Children’s Hospital in Columbus, Ohio; The Children’s Hospital at TriStar Centennial in Nashville, Tennessee; The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute in Columbus, Ohio; and University of Chicago/Comer Children’s Hospital in Chicago.
Sickle cell disease affects roughly 100,000 people in the U.S. and is more prevalent in the Black community. The only approved treatment for sickle cell had been a bone marrow transplant.
Casgevy, or exagamglogene autotemcel, works to edit genes through stem cell transplantation in patients age 12 and older. The one-time treatment takes several months and uses the patient's own blood stem cells from their bone marrow. Patients undergo chemotherapy to deplete their bone marrow to ensure no defective cells remain. Providers then extract the sickle cell gene from the patient's stem cells and reinfuse the modified cells back into the patient.
The treatment was created not to cure the disease but to reduce the number of severe health crises for the patient. Of 44 patients who received Casgevy in a trial, 29 reported having successful treatment, according to the FDA.
The FDA also approved Lyfgenia, a similar gene therapy treatment for sickle cell disease.
