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CLINICAL TRIAL OF GENE THERAPY FOR MPS VI - A SEVERE LYSOSOMAL STORAGE DISORDER
I-PRO is partner of the MeuSIX FP7 research project, under the coordination of Fondazione Theleton.
First patient was treated in October 2017.
The MeuSIX consortium planned to conduct a multicenter phase 1/2 clinical trial to investigate the safety and efficacy of AAV-mediated gene therapy in patients with MPS VI. An orphan drug designation (ODD) has been obtained from both the European Medicinal Agency and the US Food and Drug Administration for the MPS VI therapeutic AAV vector.
The results from this clinical trial proposed by the MeuSIX consortium has the potential to have a tremendous impact on the natural history of MPSVI and to significantly improve the quality of life of the affected patients. Moreover, the approach developed may facilitate the development of similar approaches for other inborn errors of metabolism.
I-PRO had the role of management of the Clinical trial.
The project, funded by the European Commission within its Seventh Framework Programme under the thematic area "Preclinical and/or clinical development of substances with a clear potential as orphan drugs" (HEALTH.2012.2.4.4-1), with Grant agreement number: 304999, has started in 2012 and has a duration of 5 +2 years, after which it has been continued by Telethon.
In 2021, after 6 years of study duration, IPRO has transferred the database to the Promoter (Telethon).
Funded by the EC, 7th Framework Programme
Fondazione Telethon, Italy
ReGenX Biosciences, USA
GenoSafe SAS, France
Federico II University of Naples, Italy
Hacettepe University, Turkey
Erasmus University, The Netherlands
Bicocca University of Milan, Italy
Informapro s.r.l., Italy