The San Raffaele Telethon Institute For Gene Therapy – Sr-Tiget (MI)

At this Institute, led by Prof. Luigi Naldini, we have been pioneers in gene therapy in Italy, delivering on promises made to the patient community. Here, in our laboratories, we have developed a therapy for ADA-SCID, with a treatment model that is saving the lives of children around the world. We are already investigating its use to treat other childhood disorders and hope that, in future, it might also be used to help patients with other diseases.

This all-Italian success story, made possible thanks to millions of Italians who support us with their donations, is giving families around the world renewed hope that their children will live a happy life.

“At the SR-Tiget Institute, we bring research from the laboratory bench to the patient’s bedside”.

Luigi Naldini, Director of SR-Tiget

The SR-Tiget Institute has always adopted a translational approach: our mission is to perform cutting-edge research and transfer the findings of laboratory research to clinical practice and thus to the patient.

To support this process, the Institute has set up two structures, each with a unique function: a GLP (Good Laboratory Practice) Test Facility for conducting pre-clinical trials in accordance with international guidelines on good laboratory practice, certified by the Italian Ministry of Health, and a Vector Integration Core for monitoring safety aspects in gene and cell therapy pre-clinical and clinical trials.

Lastly, the fact that the SR-Tiget Institute is located within the San Raffaele Hospital and works closely with its clinical units is vitally important for achieving the ultimate goal of bringing novel therapies to patients.

Personal data related to SR-Tiget research activity is managed according to a joint privacy policy signed by Fondazione Telethon and Ospedale San Raffaele.

The case of Strimvelis

Thanks to an agreement with GlaxoSmithKline, signed in 2010, the excellent results achieved by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan have been translated into a therapy that is available to all patients in Europe (the licence was transferred to Orchard Therapeutics in 2018).

The case of Libmeldy

The world's first gene therapy for metachromatic leukodystrophy, called Libmeldy, has been approved in Europe in 2020. This was announced by the Telethon Foundation, Ospedale San Raffaele and the manufacturer, Orchard Therapeutics, after the European Commission officially announced full approval.

Lines of research

The Institute conducts research projects along three main lines.

Basic research

Studies in this area are aimed at understanding the mechanisms underlying different genetic disorders, developing novel therapeutic approaches such as genome editing and designing improved cell therapies.
Heads of the research teams involved:

  • Raffaella Di Micco
  • Andrea Ditadi
  • Anna Kajaste-Rudnitski
  • Angelo Lombardo
  • Renato Ostuni

Translational research

Studies in this area are aimed at developing gene and cell therapy strategies for different genetic disorders. This line of research is primarily aimed at continuously improving the technologies used and developing methods for ensuring the safety of treatments and modulating immune responses.
Heads of the research teams involved:

  • Alessandro Aiuti (deputy director)
  • Andrea Annoni
  • Maria Ester Bernardo
  • Alessio Cantore
  • Giuliana Ferrari
  • Bernhard Gentner
  • Silvia Gregori
  • Angela Gritti
  • Eugenio Montini
  • Alessandra Mortellaro
  • Luigi Naldini (director)
  • Anna Villa

Clinical research

This area includes clinical trials for new gene and cell therapies developed at the Institute in patients with genetic disorders, in collaboration with the Haematology and Bone Marrow Transplant Unit and the Paediatric Immuno-Haematology Unit at the San Raffaele Hospital.
Heads of the research teams involved:

  • Alessandro Aiuti

The diseases we study

Immunodeficiency disorders: which?

Lysosomal storage diseases: which?

Blood disorders: which?

Bone diseases: which?

Other diseases

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