From Research
29.07.26
Epigenetic editing: a technology pioneered at SR-Tiget enters clinical trials for hepatitis B
SR-Tiget’s epigenetic editing technology can permanently silence genes without cutting DNA and has now entered clinical trials for hepatitis B.
27.07.26
ER-phagy: a new strategy to target cellular quality control
The TIGEM LEAD-ER project investigates whether ER-phagy can be pharmacologically activated to remove misfolded proteins involved in different diseases.
20.07.26
Beta-thalassaemia: from the first trial to a new gene therapy protocol
A new clinical trial is evaluating an optimised ex vivo gene therapy protocol for transfusion-dependent beta-thalassaemia developed at SR-Tiget.
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.
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.
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.
19.05.26
SR-Tiget’s Development Facilities: where gene therapy takes shape
How SR-Tiget’s development facilities are key to turn research into advanced therapies, and why they represent a new professional frontier.
