From Telethon Foundation
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.
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.
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.
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.
28.04.26
Fondazione Telethon launches Beyond, a new european postdoctoral program in advanced therapies
European Commission awards approximately €6 million under the Marie Skłodowska-Curie Actions, recognising Fondazione Telethon’s leadership in advanced cell and gene therapy research and training.
31.03.26
In memory of Claudio Bordignon, pioneer of gene therapy
His work combined scientific vision, experimental rigor and a constant focus on the potential impact of research for patients affected by severe inherited diseases.
23.03.26
Cytiva and San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) launch collaboration to advance next-generation genomic medicine platforms
New “Danaher Beacon,” first on the European continent, aims to accelerate translation, scalability, and reliability across cell and gene therapy workflows. Focus is on lentiviral gene transfer and targeted gene editing of hematopoietic stem cells.
27.02.26
Fondazione Telethon announces positive CHMP opinion recommending unlimited renewal of Strimvelis™ marketing authorisation, ten years after first approval
The ADA-SCID gene therapy, manufactured and distributed by Fondazione Telethon in the European Union since 2023, continues to demonstrate a favourable benefit-risk balance.
