Updates in the treatment of SCD and thalassemia from EHA 2024: key trial updates, challenges with gene therapies & unmet needs

Today’s VJHemOnc podcast features experts Rabi Hanna, MD, The Cleveland Clinic Taussig Cancer Institute, Cleveland, OH, Subarna Chakravorty, MBBS, MRCPCH, FRCPath, PhD, King’s College Hospital NHS Foundation Trust, London, UK, and Ali Taher, MD, PhD, FRCP, American University of Beirut Medical Center, Beirut, Lebanon. They discuss key updates in sickle cell disease (SCD) and thalassemia from the 29th Congress of the European Hematology Association (EHA), held in Madrid, Spain.

 

The experts share key trial updates, including the Phase I/II RUBY trial (NCT04853576) of renizgamglogene autogedtemcel (reni-cel) for SCD, and the Phase III ENERGIZE trial (NCT04770779), investigating the safety and efficacy of mitapivat in non-transfusion-dependent (NTD) alpha- or beta-thalassemia. They also speak on unmet needs and challenges with gene therapies for these patients.