News
19.12.25
The sustainability of gene therapies for rare diseases: a model born from ADA-SCID and WAS
Fondazione Telethon have obtained marketing authorisation for a gene therapy thanks to a sustainability model that integrates research with industry, patients and supporters. Read the full interview with Celeste Scotti, R&D Director at Fondazione Telethon.
16.12.25
From Wiskott–Aldrich Syndrome to a new model for gene therapies
Rare and ultra-rare genetic diseases pose profound scientific, human and economic challenges. The development of gene therapy for Wiskott–Aldrich syndrome (WAS) is a clear example of this complexity.
15.12.25
Fondazione Telethon and Orphan Therapeutics Accelerator Sign Memorandum of Understanding to Pioneer Non-Profit Commercial Access Model for Ultra-Rare Disease Gene Therapy in the US
Orphan Therapeutics Accelerator and Fondazione Telethon today announced the signing of a Memorandum of Understanding to facilitate US commercial access to an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome.
10.12.25
Fondazione Telethon Announces FDA approval of Waskyra™ (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome
The therapy represents a major scientific and clinical achievement, offering new hope for patients affected by this condition.
05.12.25
Engineering peripheral immune tolerance: Tregs and beyond
Tregs and beyond: discover how SR-TIGET scientists are engineering peripheral immune tolerance for tomorrow’s precision therapies.
14.11.25
CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)
Announced the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending marketing authorisation in the European Union for Waskyra™, an ex vivo gene therapy for Wiskott-Aldrich Syndrome.
13.11.25
Cracking the Code: IPEX, FOXP3 and the Path to Gene Therapy
Discover how SR-TIGET scientists advanced the genetics of peripheral immune tolerance, from FOXP3 and IPEX to gene therapy using lentiviral vectors.
04.11.25
The origins of peripheral immune tolerance: SR-TIGET's contribution to a Nobel-winning field
Discover how SR-TIGET scientists helped shape the science of peripheral immune tolerance, from the discovery of Treg and Tr1 cells to early gene therapy.
16.10.25
Attya Omer Named Runner-Up in the Nature Inspiring Women in Science Awards
The scientist of SR-TIGET and Università Vita-Salute San Raffaele recognized internationally for her vision in advancing gene therapy and inspiring women in science.
