The promise of gene and cell therapy has become a clinical reality for several devastating diseases. However, major hurdles constrain their economic sustainability and endanger their survival on the drug market, especially those for rare diseases, which calls for innovative solutions.
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C.S. is the director of research and development at Fondazione Telethon, which is the marketing authorization holder for Strimvelis. A.A. is a principal investigator of clinical trials for rare genetic diseases sponsored by Orchard Therapeutics or Fondazione Telethon. L.N. is the inventor on several patents on lentiviral vector–mediated gene therapy.
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Scotti, C., Aiuti, A. & Naldini, L. Challenges and solutions to the sustainability of gene and cell therapies. Nat Rev Genet 26, 437–438 (2025). https://doi.org/10.1038/s41576-025-00827-0
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DOI: https://doi.org/10.1038/s41576-025-00827-0
