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In memory of Claudio Bordignon

01 / 04

Fondazione Telethon mourns the passing of Professor Claudio Bordignon, a pioneer of gene therapy and the first Director of the San Raffaele Telethon Institute for Gene Therapy.

Collaboration between Cytiva and SR-TIGET

02 / 04

Objective: to develop next-generation genomic medicine platforms

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.

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).

Our numbers

Since 1990 we fund research on rare genetic conditions
  • 741 Millions of euros invested
  • 1.916 Researchers
  • 3.118 Projects funded

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