ASH 2023 | SCD highlights at ASH 2023: disease pathogenesis, ongoing clinical trials & novel gene therapies

In this discussion, Rabi Hanna, MD, Cleveland Clinic, Cleveland, OH, and Josu de la Fuente, PhD, FRCP, FRCPI, FRCPCH, FRCPath, Imperial College London, London, UK, share key highlights in sickle cell disease (SCD) presented at ASH 2023. Dr Hanna and Dr de la Fuente first discuss updates in the pathogenesis of SCD, highlighting the role of ineffective erythropoiesis. Following this, the experts summarize data emerging from the RISE UP trial (NCT05031780), which is exploring the safety and efficacy of mitapivat in patients with SCD. Dr Hanna and Dr de la Fuente then go on to discuss the excitement surrounding novel gene therapies, highlighting two agents: exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), and conclude by commenting on other gene-editing approaches being explored in the field. This discussion took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.

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