Crispr

• HCA Healthcare has announced a new study published in the New England Journal of Medicine (NEJM) highlighting advancements in CRISPR-based therapies for children. • The research builds upon pioneering work at TriStar Centennial Medical Center and the Sarah Cannon Transplant and Cellular Therapy Network, which performs over 1,600 cellular therapies and transplants annually. • Dr. Frangoul, a key investigator, previously contributed to the first U.S. clinical trial for gene-editing in sickle cell disease, leading to the first FDA-approved CRISPR therapy.

• The FDA granted accelerated approval to a CRISPR-based gene therapy for severe sickle cell disease on April 20, marking the first approved genetic treatment editing patient blood cells to produce functional hemoglobin. • Clinical trials demonstrated 95% of treated patients experienced elimination or near-elimination of vaso-occlusive crises within 12 months, with durable responses observed for over two years post-treatment. • The therapy addresses a disease affecting approximately 100,000 Americans, primarily of African descent, offering potential cure for patients previously reliant on pain management and blood transfusions.