News
24.04.25
Clinical Evidence Confirms Timing as Crucial in Gene Therapy for MLD
A NEJM study confirms that gene therapy, developed over 20 years at San Raffaele-Telethon, preserves motor and cognitive function in MLD patients if administered early.
31.03.25
From psoriasis to Lowe syndrome: clinical trial kicks off thanks to collaboration with Can-Fite
Research conducted at TIGEM has identified a molecule able to counteract renal dysfunction in this rare syndrome of genetic origin.
27.03.25
Research, innovation and global success: EsoBiotec takes off also thanks to Italian science
From innovation to value creation: the crucial role played by the research performed at OSR and Fondazione Telethon in the development of innovative technologies, has been confirmed by the success of EsoBiotec SA.
11.03.25
BLA for the gene therapy for the treatment of Wiskott-Aldrich syndrome submitted to FDA
The Biologic License Application for the gene therapy for the treatment of the Wiskott-Aldrich Syndrome (WAS) has been submitted to the Food and Drug Administration
03.03.25
A platform dedicated to Duchenne Muscular Dystrophy
Created thanks to the contribution of some companies, it will make available the data of hundreds of DMD patients and will lay the foundations for collecting new data in the future according to homogeneous standards.
03.02.25
MAA for the Gene Therapy for the treatment of Wiskott-Aldrich Syndrome submitted to EMA
The Marketing Authorization Application for the gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS) has been submitted to the European Medicines Agency
03.12.24
ERC Consolidator Grants Awarded to Two SR-Tiget Researchers
Daniela Cesana and Alessio Cantore presented two projects, respectively to investigate the impact, safety and efficacy of gene therapy through the 'signatures' of circulating DNA and to investigate the biology of liver cells in order to exploit them in the field of gene therapy.
28.11.24
Gene therapy: target liver
At the San Raffaele-Telethon Institute in Milan, an innovative liver-directed gene transfer platform applicable to various metabolic diseases has been developed thanks to the efforts of the research group led by Alessio Cantore, together with the biotech company Genespire.
18.11.24
Mucopolysaccharidosis 6 and gene therapy: present and future
A study just published by TIGEM researchers shows how the efficacy of this experimental therapy is maintained over time, at the highest doses. Study coordinator Brunetti-Pierri reports on possible future developments
