From Research
03.08.26
Working at the intersection of Biology and Data Science at TIGEM
At TIGEM, biologists, bioinformaticians and statisticians combine data, computational models and laboratory research to advance rare disease science.
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.
