News
13.07.26
A new gene therapy clinical trial launched for patients with transfusion-dependent beta-thalassemia
Fondazione Telethon and IRCCS Ospedale San Raffaele, sponsors of the trial, together with Ospedale Pediatrico Bambino Gesù launch a multicenter study based on an optimized gene therapy protocol developed at the SR-Tiget Institute.
08.07.26
Genespire and SR-Tiget show durable preclinical efficacy of liver-directed gene therapy for methylmalonic acidemia
The spin-off of Fondazione Telethon and Ospedale San Raffaele reports positive results in preclinical models of this rare metabolic disorder, further supporting the path toward first-in-human application.
02.07.26
Fondazione Telethon to lead €30 million ERDERA European call
The initiative, dedicated to funding clinical studies on rare diseases, will include active patient organization involvement at every stage of research.
23.06.26
ESMRank: using AI to predict the effects of protein mutations
At TIGEM, ESMRank uses artificial intelligence to rank protein mutations and help researchers interpret genetic variants linked to disease.
17.06.26
Doing a Postdoc in Advanced Therapies: why choose SR-Tiget?
At SR-Tiget, Postdocs are trained through excellence in research, a translational vision, mentorship, and international opportunities.
15.06.26
Fondazione Telethon appoints Graciana Diez-Roux as new Scientific Director
She will define the Foundation’s scientific strategy to further advance rare genetic disease research and support the next generation of scientists.
10.06.26
TUDP: genomics, bioinformatics and knowledge for undiagnosed diseases
Fondazione Telethon’s TUDP combines genomics, bioinformatics and research to identify the genetic causes of rare paediatric diseases without a diagnosis.
03.06.26
New “SMArT” platform makes gene editing in hematopoietic stem cells more efficient and safer
Nature Biotechnology study shows near-pure selection of correctly edited blood stem cells while reducing dangerous genomic alterations.
29.05.26
Fondazione Telethon advances national Duchenne registry for research and regulation
A nationwide network of 16 Italian neuromuscular centers and over 1,200 patients to support research and new therapies.
