Ensuring a future for gene therapy for rare diseases. Comment from Alessandro Aiuti francescapasi and NaldiniLab San Raffaele Telethon Institute of Gene Therapy, Italy
A.A. is PI of clinical trials sponsored by Orchard Therapeutics, which licensed gene therapy products for ADA-SCID, WAS, metachromatic leukodystrophy , β-thalassemia and mucopolysaccharidosis type I originally developed at SR-Tiget. A.A. is a member of the Committee for Advanced Therapies and his views are personal and may not be understood or quoted as being made on behalf of the European Medicines Agency . L.N.
is an inventor on pending and issued patents on LV technology and gene editing filed by the Salk Institute, Cell Genesys, Telethon Foundation and/or San Raffaele Scientific Institute. L.N.
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