On the eve of World Rare Disease Day, the Telethon Foundation announces its commitment to making gene therapy for Wiskott-Aldrich syndrome, a rare genetic disease of the immune system, soon available.
This is the second stage of a path traced by Telethon to prevent gene therapies, potentially capable of changing the natural history of very serious diseases, from being unavailable to patients.
Already last September, the Foundation announced that it had assumed the responsibility of producing and distributing gene therapy for another rare immunodeficiency, ADA-SCID, which despite the confirmation of its efficacy and safety risked no longer being available.
Now it is the turn of gene therapy for Wiskott-Aldrich syndrome, which affects almost exclusively males and manifests itself from early childhood with recurrent and relapsing infections, bleeding, eczema, increased risk of developing autoimmune diseases and lymphomas.
This therapy was also born in the laboratories of the San Raffaele-Telethon Institute in Milan and was subsequently the subject of an industrial partnership.
In 2022 the pharmaceutical company that had it under license announced its disinvestment in the field of immunodeficiencies, before requesting marketing authorization from the EMA, the European Medicines Agency.
Telethon Foundation therefore obtained the return of the license and took on the challenge of obtaining approval for this therapy from regulatory bodies.
EMA has selected Telethon and the development program of this therapy for its pilot acceleration project, which supports academic institutions and non-profit organizations in the development of advanced therapies.
The Foundation will be supported through a rigorous process of regulatory consultancy and pre-authorisation checks in the application for marketing in Europe.
"After the decision taken for gene therapy for ADA-SCID - explains Francesca Pasinelli, managing director of the Telethon Foundation - we want to guarantee the availability of another treatment which would otherwise risk failure to access the market".
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