Director: Luigi Naldini
Deputy Director: Alessandro Aiuti
The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), directed by Luigi Naldini, is a joint-venture between the San Raffaele Scientific Institute and the Telethon Foundation. The mission of the Institute is to perform innovative research on gene transfer and cell transplantation and translate its results into successful clinical application of gene and cell therapy for genetic diseases.
The research activities of the Institute span from basic to early phase clinical studies aiming to:
i) identify the genetic bases and the pathophysiological processes of the inherited diseases under study, which include primary immunodeficiencies, blood diseases, inherited leukodystrophies and other lysosomal storage diseases;
ii) develop new gene transfer and editing technologies for more efficient and safe genetic correction of diseases using ex vivo and in vivo strategies;
iii) characterize the biological properties of hematopoietic and neural stem cells and establish procedures for their ex vivo manipulation and transplantation;
iv) investigate cell types mediating innate and adaptive immunity and modulate immune response to gene and cell products to improve efficacy and stability of the therapy;
v) test the new therapeutic strategies in preclinical disease models instrumental to design clinical trials;
vi) exploit the technologies and models developed above to design new gene therapy strategies for cancer.
All these goals are fulfilled within the SR-Tiget Experimental Research Units.
vii) assess the efficacy and safety of the novel therapeutic strategies in patients within early phase clinical trials. This clinical activity is conducted within the SR-Tiget Clinical Research Unit (CRU; Clinical Research Coordinator: Alessandro Aiuti), which is devoted to the diagnosis, treatment and follow-up of patients with primary immunodeficiencies, hematologic and metabolic diseases, and comprises the SR-Tiget Clinical Trial Office (TCTO). The CRU works in close collaboration with the Pediatric Immuno-Hematology Unit (directed by Alessandro Aiuti) and the Hematology and Bone Marrow Transplantation Unit (directed by Fabio Ciceri) of the San Raffaele Hospital.
Moreover, SR-Tiget includes the Vector Integration Core, aimed at monitoring and profiling vector integration genome-wide in preclinical and clinical studies, and a GLP test facility, aimed at conducting preclinical studies of gene and cell therapy according to GLP (Good Laboratory Practice) guidelines.
The gene and cell therapy products used in the current clinical trials are produced in the GMP (Good Manufacturing Practice) facility of MolMed S.p.A.
Overall, this research environment provides world-recognized leading scientific expertise in gene transfer and editing technologies, genetic modification of stem cells, preclinical models of stem cells transplantation, as well as in the translational, regulatory and clinical issues associated with testing novel cell and gene therapies for immune, hematological and lysosomal storage diseases.