News
30.07.25
Usher Syndrome: Promising Preliminary Results from Gene Developed by TIGEM
An experimental gene therapy developed at the Telethon Institute of Genetics and Medicine (TIGEM) in Pozzuoli has shown encouraging clinical results in patients with Usher syndrome type 1B. The first data, presented one year after the start of the clinical trial, show a significant improvement in visual capacity.
17.06.25
ERC Awards €2.5M to TIGEM scientist for project on programmable genetic circuits
di Bernardo’s DIMERCIRCUITS integrates AI and synthetic biology to advance precision gene therapy.
16.06.25
Andrea Ballabio awarded Beth Levine Prize in Autophagy Research from UT Southwestern
Andrea Ballabio, founder and former director of the Telethon Institute of Genetics and Medicine (TIGEM) and Professor of Medical Genetics at the University of Naples “Federico II” has been named the recipient of the 2025 Beth Levine, M.D. Prize in Autophagy Research from UT Southwestern Medical Center.
24.04.25
MLD and gene therapy: a game-changing treatment backed by NEJM—Timing Is Everything
The study was conducted at Ospedale San Raffaele in Milan and is the result of more than 20 years of research by the San Raffaele-Telethon Institute for Gene Therapy and the commitment of Fondazione Telethon to transforming research into therapies available for patients.
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).
