Positive CHMP opinion: unlimited renewal of Strimvelis™

01 / 04

Among the very first gene therapies ever made available as a medicinal product, it is currently the longest-marketed gene therapy available on the market.

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Tigem: technological platforms serving research

02 / 04

Tigem’s core facilities combine advanced technology and scientific mentoring, accelerating growth and research for PhD students and postdocs.

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Gene therapy platform for lysosomal storage diseases

03 / 04

A new gene therapy platform for lysosomal storage diseases enables parallel development, reducing time, costs and improving sustainability for rare disease treatments.

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Announced FDA approval of Waskyra™

04 / 04

Fondazione Telethon announces that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex vivo gene therapy for patients with Wiskott-Aldrich syndrome (WAS).

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Our numbers

Since 1990 we fund research on rare genetic conditions

741 Millions of euros invested
1.916 Researchers
3.118 Projects funded

What we do

Our mission

We are an Italian non-profit organization committed to advancing research on rare and complex genetic diseases. For over 35 years, we have supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions.

Institutes

We established two Institutes in Italy, now internationally recognized as centers of excellence: The San Raffaele Telethon Institute For Gene Therapy in Milan and Telethon Institute of Genetics and Medicine in Pozzuoli (NA).

Research initiatives

We drive excellence and innovation in research on rare genetic diseases, building a pipeline that spans from basic science to clinical trials and approved therapies.

The sustainability of gene therapies for rare diseases: a model born from ADA-SCID and WAS

Storie e News

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.

News

Read all the news

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.

From Research

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.

From Research

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.

From Telethon Foundation

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