About us

Our Lab works in close interaction with the Cancer Gene Therapy Unit lead by Prof. Luigi Naldini, at the IRCCS Ospedale San Raffaele, at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET).

Luigi Naldini

Luigi Naldini, M.D., Ph.D., is the Scientific Director of the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine.

A study lead by Luigi Naldini was the first to describe engineering of lentivirus to stably transfer genetic material to target cells. Thereafter, Luigi Naldini’s team has pioneered applications of lentiviral vectors for gene transfer in biomedical research. A lentiviral vector-based drug conceived and developed at SR-TIGET has now been approved for clinical usage and others are being tested in clinical trials on several untreatable and otherwise deadly human diseases, including cancer. Luigi Naldini has also pioneered the use of endonucleases to perform targeted DNA insertion of genetic material and gene editing.

Luigi Naldini has been awarded the Jimenez Diaz Prize in 2016, the Beutler Prize from the American Society of Hematology (ASH) in 2017 and the 2019 Jeantet-Collen Prize for Translational Medicine. He was nominated “Grande Ufficiale dell’Ordine Al Merito della Repubblica Italiana”, one of the highest-ranking honors in Italy, from the President of the Republic and the Prime Minister of Italy.

IRCCS Ospedale San Raffaele

IRCCS Ospedale San Raffaele is a university and research hospital established in 1971 to provide specialized care for the most complex health conditions and to perform cutting-edge translational research. 

Thanks to 100 high-tech laboratories and pre-clinical facilities, 50 medical specialties, 50.000 hospitalizations per year, 2.081 peer-reviewed publications in 2020 alone and more than 880 ongoing clinical trials, at Ospedale San Raffaele basic science translate quickly into clinical practice and clinical needs drive basic research. 

Among the principal area of investigation: oncology, immunology, infectious diseases, neuroscience, advanced imaging and genetics. The institute is recognized as a global authority in gene and cell therapies.


San Raffaele Telethon Institute for Gene Therapy

The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) was created in 1995 as a joint-venture between Fondazione Telethon and Ospedale San Raffaele  (OSR), with the mission to perform cutting edge research on gene and cell therapy and to translate its results into therapeutic advances for genetic diseases.

Over the years, SR-Tiget has given a pioneering contribution to the gene and cell therapy field with relevant discoveries in vector design, gene transfer and gene editing strategies, stem cell biology, identity and mechanism of action of regulatory cells in immune responses and innate immune cells in cancer. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and lysosomal storage disorders, which have already treated 121 patients and led to the filing and approval of novel advanced therapy medicines. More recently SR-Tiget has also embarked on developing applications of its cell and gene therapy platforms to the treatment of certain types of cancer.

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People from 10 distinct countries


Publications 2018 – 2022 with a mean IF of 11.934


Active patents and/or patent applications


Patients treated with gene therapy  products

  • Leadership in lentiviral gene transfer technology
  • Robust translational capacity in developing applications of gene transfer and gene editing to the development of novel gene and cell therapies
  • State-of-the-art stem cell transplant center
  • Pioneering successful first-in-human phase I/II gene therapy trials
  • In house facilities & resources supporting
    • translation research (GLP Test Facility, Process Development Lab)
    • early phase cell and gene therapy clinical trials (Clinical Research Unit, Clinical Trial Office, Regulatory Affairs Office, GCLP Lab, Vector Integration Core)
  • Strategic alliances with pharma and biotech
  • Roster of new PI’s venturing into relevant new technologies and emerging biological concepts

Contact our team

Targeted Cancer Gene Therapy Unit
San Raffaele Telethon Institute for Gene Therapy (SR-TIGET)
IRCCS Ospedale San Raffaele 
Via Olgettina 58, 20132 Milan

email: squadrito.mario@hsr.it

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