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SR-Tiget is a world-leading joint venture between Ospedale San Raffaele and Fondazione Telethon, dedicated to performing cutting-edge research in gene and cell therapy. Based in Milan, Italy, the institute has pioneered the use of lentiviral vectors for hematopoietic stem cell (HSC) gene therapy. Their work led to the development of the first-ever ex vivo gene therapy, Strimvelis, and the more recent Libmeldy for metachromatic leukodystrophy. SR-Tiget focuses on translating basic research into clinical treatments for rare genetic diseases and cancer. The institute maintains significant partnerships with global pharmaceutical companies to commercialize its innovative therapeutic pipeline.
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