Company intelligence / Profile preview

TIGET

Milan, Lombardy, Italy
Ownership
Private
Employees
~200
Development stage
Other
01

Overview

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.

Therapeutic areas
HematologyImmunologyNeurologyRare Diseases
Modalities
Gene therapyCell therapyLentiviral vectorHSC gene therapyCRISPR-based therapy
Industry
Academic Hospital/University/Research
02

Partnerships

GSKOrchard TherapeuticsRocket PharmaceuticalsGenenta Science

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