News
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.
23.02.26
AAV gene therapy for large genes: two TIGEM platforms
How AAV gene therapy for large genes overcomes size limits through two TIGEM technology platforms for inherited retinal and rare diseases.
11.02.26
February 11: The Value of Women in Research
Bioinformatics and translational research at TIGEM, as told by two of its leading women researchers, on the International Day of Women and Girls in Science.
05.02.26
Gene therapy platform for lysosomal storage diseases
A new gene therapy platform for lysosomal storage diseases enables parallel development, reducing time, costs and improving sustainability for rare disease treatments.
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.
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.
