News
16.06.25
Andrea Ballabio awarded Beth Levine Prize in Autophagy Research from UT Southwestern
Andrea Ballabio has been named the recipient of the 2025 Beth Levine, M.D. Prize in Autophagy Research from UT Southwestern Medical Center.
03.06.25
Long‑term dysfunction of edited blood stem cells and how to overcome it
SR‑Tiget researchers identify premature aging in CRISPR‑edited blood stem cells, but show that targeted interventions such as p53 inhibition and Anakinra can restore long‑term regenerative function.
28.05.25
A new window for in vivo blood‑stem‑cell gene therapy
A study from SR Tiget shows that blood stem cell gene therapy can be delivered directly in newborns during a short early life window.
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
