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J.B. is a consultant to and has licensed advanced therapy medicinal products to Rocket Pharma. A.A. is principal investigator of clinical trials sponsored by Orchard Therapeutics, which has licensed gene therapy products for ADA-SCID, Wiskott-Aldrich syndrome, metachromatic leukodystrophy, β-thalassemia and mucopolysaccharidosis type I, originally developed at SR-Tiget, and is a member of the Committee for Advanced Therapies; his views are personal and may not be understood or quoted as being made on behalf of the European Medicines Agency. A.L. is principal investigator of an academic clinical trial for RAG-1 SCID with a licensing agreement to Mustang Bio. C.B. is an investigator of clinical trials sponsored by Rocket Pharma, Bluebird Bio, AvroBio and previously Orchard Therapeutics in addition to academic clinical trials.
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Fox, T., Bueren, J., Candotti, F. et al. Access to gene therapy for rare diseases when commercialization is not fit for purpose. Nat Med 29, 518–519 (2023). https://doi.org/10.1038/s41591-023-02208-8
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DOI: https://doi.org/10.1038/s41591-023-02208-8
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