Osteopetrosis: new opportunity of treatment thanks to a licensing agreement

SiSaf announces a research collaboration and licensing agreement with the University of L’Aquila for the treatment of the debilitating bone disease, Autosomal Dominant Osteopetrosis Type 2.

From Research


Discovery of a novel drug candidate to develop effective treatments for brain disorders

Researchers at IIT-Istituto Italiano di Tecnologia discovered a novel chemical compound, which has the potential to became a new drug for the treatment of core symptoms of brain disorders like Down syndrome and autism.

From Research


Study of genetic condition leads to discovery of mechanism that promotes the formation of renal cysts and tumours, thanks to the support of Telethon and AIRC

At the Telethon Institute of Genetics and Medicine (Tigem) in Pozzuoli, the team led by Andrea Ballabio has described in Nature* how the study of a rare genetic condition has provided the key to understanding a mechanism that leads to the formation of cysts and tumours in certain organs, in particular the kidneys. Supported by Fondazione Telethon, Fondazione AIRC for cancer research and Regione Campania, the study included the participation the European Institute of Oncology (IEO) and the Institute of Cell Biology at the University of Innsbruck

From Research


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

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