Regenerative medicine and gene therapy

The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) was created in 1995 as a joint venture between the Telethon Foundation 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.
Overall, 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, which possess 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.

ORGANIZATION
SR-Tiget is organized into 15 research units (14 basic research units and 1 clinical research units) with a total number of over 150 scientists.

BASIC RESEARCH
Research at SR-Tiget spans from basic research to pre-clinical studies to early phase clinical trials according to the following major aims:

  • identifying the genetic bases and the pathophysiological processes underlying several types of inherited diseases, including for autoimmune diseases, hematologic diseases, inherited leukodystrophies and neurodegenerative diseases;
  • developing novel gene and cell therapy strategies;
  • ameliorating the performance and safety of gene transfer employed for these therapies;
  • developing novel technological platforms, including targeted genome and epigenome editing;

CLINICAL RESEARCH
SR-Tiget portfolio of gene and cell therapies now embraces the full spectrum of drug development up to the market. Notably, in May 2016 the European Commission granted marketing authorization for Stimvelis for the treatment of ADA-SCID, a severe form of immunodeficiency. Strimvelis, which is the first approved ex vivo gene therapy worldwide, has been developed at SR-Tiget and brought to the market under a strategic alliance with GSK. The successful results obtained with ADA-SCID provided a rationale for extending the HSC gene therapy approach to other diseases. In particular, Wiskott-Aldrich Syndrome (WAS) and Metachromatic Leukodystrophy (MLD), beta‐thalassemia, and  Mucopolysaccharidosis type I (MPS-I) is planned to start in 2018.

More info can be found here.