Dr. Rabi Hanna from Cleveland Clinic shares insights on innovative treatments for severe sickle cell disease. He presents updated results from the RUBY trial, highlighting the potential of reni-cel as a groundbreaking gene therapy. The conversation also touches on the promising outcomes of the HIBISCUS trial for Etavopivat, showing significant improvements in patient quality of life. Furthermore, advancements in thalassemia treatments are discussed, focusing on reduced transfusion needs from the ENERGIZE-T study.

Leading experts Rabi Hanna, MD and Josu de la Fuente, PhD discuss key updates in sickle cell disease at ASH 2023. They cover topics such as disease biology, ongoing trials, and the growing role of gene therapy. They explore novel agents, pathogenesis, and potential therapies, highlighting improvements in natural history, mortality rates, and access to healthcare. The speakers also discuss advancements in treatment, including the reduction in vasoclecive events and hospitalizations, and the paradigm shift towards increasing fetal hemoglobin for management.