News
07.09.20
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.
10.07.20
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.
01.07.20
Study of genetic condition leads to discovery of mechanism that promotes cance development
At TIGEM scientist have described 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.
04.06.20
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.
20.04.20
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.
11.04.19
Gene Therapy Demonstrates Efficacy in Treating Wiskott-Aldrich Syndrome
A clinical study confirms the efficacy of gene therapy as a treatment for Wiskott–Aldrich Syndrome (WAS), a serious rare genetic disease affecting blood cells.
21.01.19
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.
14.11.18
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.
21.06.16
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).
