Groundbreaking Gene Therapies for Hereditary Diseases

Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Kröner Fresenius Prize for Medical Research 2020 endowed with 2.5 million euros.

From Telethon Foundation


Sofinnova Partners Announces Third Investment from its Italian Fund

This investment brings the total financing by Sofinnova Partners to €25 million over the past six months.

From Telethon Foundation


Tigem and SR-Tiget involved in UPGRADE project, funded by the European Union

Fondazione Telethon announces the launch of the UPGRADE project, funded by the European Union with 15 million euros, aiming at developing more precise and effective gene therapies approaches.

From Telethon Foundation


Beta Thalassemia: clinical trial provides encouraging evidence for efficacy of gene therapy

Gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia, a genetic disease that is quite common in Mediterranean countries, affecting over 7000 patients in Italy only.

From Research


A new ingredient to potentiate gene therapy in stem cells

A new study published by the research team led by Anna Kajaste-Rudnitski at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, shows that a naturally occurring compound significantly increases the efficiency of lentiviral vector-mediated gene transfer in blood stem cells.

From Research


Restore Health – Therapies that cure

The European Commission (EC) announced that the FET Flagship RESTORE, which was initiated by the Berlin-Brandenburg Center for Regenerative Therapies (BCRT), has been selected to enter the next phase of the FET Flagship competition.

From Telethon Foundation


From epigenetics - new molecular switches to silence genes

It is possible to silence a desired gene by hiding it from the molecular machinery that has the task of reading it and expressing its function. This is the result of a research carried out at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget).

From Research


Editas Medicine Announces Scientific Multi-Year Collaboration with Fondazione Telethon and Ospedale San Raffaele

Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, and Fondazione Telethon and Ospedale San Raffaele, have entered into a scientific collaboration to research and develop genome edited hematopoietic stem cell (HSC) and T cell therapies.

From Telethon Foundation


Preliminary results reporting the safety and effectiveness of gene therapy in patients with metachromatic leukodystrophy published in The Lancet

The gene therapy developed in the laboratories of the San Raffaele Telethon Institute for Gene Therapy in Milan (SR-Tiget) remains effective as a potential early treatment of metachromatic leukodystrophy (MLD).

From Research

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