News
31.03.25
From psoriasis to Lowe syndrome: clinical trial kicks off thanks to collaboration between Fondazione Telethon and Can-fite
Research conducted by Antonella De Matteis 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
Fondazione Telethon submits US marketing authorization application for etuvetidigene autotemcel gene therapy for the treatment of Wiskott-Aldrich syndrome
Fondazione Telethon announced that it has submitted the Biologic License Application (BLA) for the gene therapy - etuvetidigene autotemcel - for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the Food and Drug Administration (FDA).
03.03.25
Fondazione Telethon launches a platform dedicated to Duchenne Muscular Dystrophy
Created thanks to the contribution of some of the most important companies involved in the development of therapies for the disease, 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
Fondazione Telethon submits EU marketing authorization application for Etuvetidigene Autotemcel Gene Therapy for the treatment of Wiskott-Aldrich Syndrome
Fondazione Telethon announced that it has submitted the Marketing Authorization Application (MAA) for the gene therapy - etuvetidigene autotemcel - for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the European Medicines Agency (EMA).
03.12.24
ERC: two researchers from San Raffaele-Telethon institute for gene therapy in Milan each receive 2 million euros for their prestigious funding together with IRCCS Ospedale San Raffaele and Università Vita-Salute San Raffaele
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
16.09.24
Naples, world's first genere therapy for a rare retinal disease
The surgery, performed at AOU Vanvitelli, was made possible by an innovative gene therapy platform developed by Pozzuoli's TIGEM that allows for large gene transfer.
