Advances in gene-based therapies for SCD and thalassemia: access, outcomes & innovation

This VJHemOnc podcast explores the latest advances in gene-based therapies for sickle cell disease (SCD) and thalassemia, highlighting progress in patient access, selection, treatment delivery, and clinical outcomes. Sunil Gupta, MD, FRCP, FRCPath, MBA, Lewisham and Greenwich NHS Trust, London, UK, discusses expanding access to gene therapy for patients with SCD, while Alexander Ngwube, MD, Baylor College of Medicine, Houston, TX, shares insights into real-world patient selection and decision-making. Rabi Hanna, MD, Cleveland Clinic, Cleveland, OH, outlines key considerations for selecting patients with SCD and thalassemia for gene therapy, discusses strategies to optimize the treatment pathway from stem cell mobilization to long-term follow-up, and reviews results from the RUBY trial (NCT04853576) of gene editing therapy in severe SCD. Zachary Crees, MD, Washington University School of Medicine, St. Louis, MO, also highlights emerging mobilization approaches, including motixafortide-based strategies to improve stem cell collection for gene therapy.