FDA Approves First Gene Therapy Treatments for Sickle Cell Disease

Published 2023-12-09

On December 8th, 2023, FDA approved two gene therapies for Sickle Cell Disease: Casgevy and Lyfgenia. Casgevy utilizes the CRISPR gene editing tool as a cell based therapy for the patients Hematopoietic Stem Cells. With Lyfgenia, the patient’s blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A. Both products are made from the patients’ own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant.