Study offers real-world data on commercial implementation of gene therapies for sickle cell disease and beta thalassemia
(ORLANDO, Dec. 6, 2025) The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers prepare to meet growing demand for gene therapies in the coming years.
“Gene therapy requires system-level coordination and close collaboration across patients, treatment centers, payers, and manufacturers,” said study author Joanne Lager, MD, chief medical officer at Genetix Biotherapeutics Inc. “The demand for these one-time durable gene ...