From Telethon Foundation
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.
16.01.26
Fondazione Telethon receives european marketing authorisation for Waskyra™ (etuvetidigene autotemcel), a gene therapy for the treatment of Wiskott-Aldrich syndrome
The European Commission’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025. The therapy was also approved by the U.S. FDA for the U.S. market in December 2025.
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.
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.
16.06.25
Andrea Ballabio awarded Beth Levine Prize in Autophagy Research from UT Southwestern
Andrea Ballabio has been named the recipient of the 2025 Beth Levine, M.D. Prize in Autophagy Research from UT Southwestern Medical Center.
11.03.25
BLA for the gene therapy for the treatment of Wiskott-Aldrich syndrome submitted to FDA
The Biologic License Application for the gene therapy for the treatment of the Wiskott-Aldrich Syndrome (WAS) has been submitted to the Food and Drug Administration
