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ASH 2023 | Thalassemia highlights at ASH 2023: novel therapies, trial updates & the value of precision medicine

In this discussion, Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, and Kevin Kuo, MD, MSc, FRCPC, University Health Network, Toronto, Canada, share key highlights in thalassemia presented at ASH 2023. Dr Cappellini and Dr Kuo first discuss the promise of novel gene therapies in thalassemia, highlighting the recent approval of two of these agents in the sickle cell space. Following this, the experts discuss updates with the use of luspatercept in thalassemia, summarizing data from the BEYOND trial (NCT03342404), and comment on other disease-modifying agents being explored. To conclude, Dr Cappellini and Dr Kuo share insights into the challenges of treating patients with thalassemia, the limitations of gene therapy in certain patient populations, and the value of precision medicine. This discussion took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Honoraria,: Bristol Myers Squibb, Sanofi, Vertex, Agios
Research Funding: Bristol Myers Squibb, Agios
Board of Directors/Advisory Committee: Sanofi, Vertex
Travel support: Sanofi
Research Funding: Sanofi, Vertex, Silence Therapeutics

Consultancy: Agios Pharmaceuticals, Alexion Pharmaceuticals, Bristol Myers Squibb, Forma Therapeutics, Pfizer, Novo/Nordisk, Vertex Pharmaceuticals
Research Funding: Agios Pharmaceuticals
Honoraria: Bristol Myers Squibb, Novo/Nordisk
Board of Directors/Advisory Committee: Bioverativ, Sanofi, Sangamo