News
04.11.26
Lowe syndrome: from basic research to the first clinical trial
After more than twenty years of research into the cellular mechanisms underlying Lowe syndrome, a compound is moving into clinical testing for this rare genetic disease.
03.11.26
Ten years of “Just like Home”: supporting families throughout the treatment journey
Launched in 2016, “Just like Home” has so far supported 140 patients through a long and complex journey that, for many, begins thousands of kilometers from home.
22.10.26
Tigem contributes to Carbon-X, the Open-Source AI Model helping decode the human genome
The Telethon Institute of Genetics and Medicine (TIGEM) has contributed to the development of a new open-source artificial intelligence model designed to make advanced genomic analysis and DNA interpretation tools more accessible to researchers worldwide.
19.10.26
Luigi Naldini elected to the U.S. National Academy of Medicine
The prestigious recognition honors a pioneer of lentiviral gene therapy whose discoveries have led to approved treatments for rare diseases, including the first gene therapy brought to the U.S. market through a non-profit development pathway.
30.09.26
Newly discovered pathway unlocks the therapeutic potential of TFEB
A study published in Nature reveals druggable regulators of TFEB, a key controller of cellular recycling, opening new opportunities for the development of therapies for rare genetic diseases, cancer and other disorders linked to lysosomal dysfunction.
29.09.26
New gut-liver link discovered in alpha-1 antitrypsin deficiency
A study led by Nunzia Pastore at TIGEM shows that the mutated protein responsible for the disease also alters intestinal cells, opening new avenues for identifying at-risk patients and understanding the variability of the disease.
28.09.26
UCSF Benioff Children’s Hospitals Become the First US Qualified Treatment Center Contracted to Administer Individualized Gene Therapy WASKYRA®
WASKYRA®, the first gene therapy approved in the US to treat Wiskott-Aldrich Syndrome, is now commercially available, with more centers expected to be added in the coming months.
24.09.26
Wiskott-Aldrich Syndrome: Long-Term Results of Gene Therapy Developed at the San Raffaele-Telethon Institute Published in the “New England Journal of Medicine“
The publication consolidates more than two decades of research and clinical development and confirms the long-term clinical benefit of an autologous gene therapy for Wiskott-Aldrich syndrome, a rare genetic disorder affecting the immune system and platelets.
18.09.26
In vivo lentiviral gene therapy for methylmalonic acidemia moves towards the clinic
GENE202 is an in vivo lentiviral gene therapy for methylmalonic acidemia developed by SR-Tiget and Genespire and now moving towards clinical testing.
