From Telethon Foundation
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.
12.03.26
Reaching the CNS with lentiviral gene therapy
SR-TIGET ex vivo and in vivo lentiviral gene therapy aims to target the CNS, building on MLD success and expanding toward new therapeutic strategies.
09.03.26
Tigem: technological platforms serving research
TIGEM’s core facilities combine advanced technology and scientific mentoring, accelerating growth and research for PhD students and postdocs.
02.03.26
Retinal dystrophies: gene-agnostic therapy based on miRNAs
New miR-based gene-agnostic strategies target shared mechanisms in retinal dystrophies, opening new therapeutic options beyond single-gene therapy.
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.
