San Raffaele Telethon Institute for Gene Therapy

San Raffaele Telethon Institute for Gene Therapy



Luigi Naldini


Deputy director clinical research

Alessandro Aiuti


The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) was created in 1996 as a joint venture between the Fondazione Telethon and Ospedale San Raffaele, with the mission to perform cutting-edge research in gene and cell therapy and to translate its results into therapeutic advances, focusing on genetic diseases.

SR-Tiget represents a multi-disciplinary research environment, which provides a unique blend of scientific expertise in the development of innovative gene and cell therapy strategies, access to relevant preclinical models to evaluate their efficacy and safety, as well as competence in conducting early phase clinical trials. This provides a fertile ground for alliances with industrial partners and launch of start-up companies, which are crucial to secure the skills and resources required to address the regulatory hurdles and manufacturing needs to bring new therapies to registration and make them available to patients.

SR-TIGET graph

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We employ a basic-to-translational, bench-to-bedside R&D approach encompassing the characterization of biological properties and physiopathological processes, the design and optimization of safety and efficacy of novel gene and cell therapy platforms and their development from preclinical models to first-in-human testing.

GOAL 1: Advance HSC gene therapy to standard-of-care for rare genetic diseases | Discover more

GOAL 2: Be at the forefront of gene editing development | Discover more

GOAL 3: Broaden application of lentiviral vectors to in vivo gene therapy | Discover more GOAL 4: Devise new cell and gene therapy strategies to modulate immune response | Discover more

Recognized assets of the Institute include:

  • Leadership in lentiviral gene transfer technology
  • Pioneering contribution to the genome and epigenome editing field
  • State-of-the-art stem cell transplantation center for adult and pediatric patients
  • In house facilities and resources supporting translational research and first-in-human phase I/II gene therapy trials
  • Strategic alliances with industrial partners and successful launch of startup biotech companies
  • Roster of young PIs venturing into relevant new technologies and emerging biological concepts