CMT Gene Therapy

Published on November 19th, 2020

European Associations’ support to research on CMT

We are proud to give the news of a fruitful synergy among the European CMT Federation members to support CMT research.

In June 2020, the Italian association ACMT-Rete and the French association CMT-France, both founding members of the ECMTF, officially supported the Mygenter gene therapy project in the scope of the EJP RD call for proposal 2020.

The Mygenter project is a gene therapy using a viral vector to introduce gene expression modifying tools directly in the target cell, configuring itself as an attractive alternative to pharmacological treatments. The goal is to pave the way to the first myelin-target gene therapy clinical trial for CMT1A.

The Mygenter researchers, for the part of the research concerning the nerves and sense organs, cooperate with Invivex, a preclinical Contract Research Organization (CRO) specialized in the evaluation of sensory and neuromotor system disorders.  

In July 2020, the Spanish association El Motor de tus Pasos, a new member of the ECMTF, raised funds for research on CMT, organizing bicycle racing throughout Spain. Despite the restrictions due to the Coronavirus pandemic, El Motor de tus Pasos succeeded in raising 15.000 €, deciding to allocate 10.000 € to Invivex and the Mygenter project.

This contribution is vital for researchers, allowing them to progress in the anti-demyelinating vector study while waiting for the EJP RD decision on the project’s financing. Many thanks to all the associations involved in this project, which believe in gene therapy’s potential and support it both with trust and financial help.

The role of the European CMT Federation was equally decisive for communication and the passing of information.

This project is only in its infancy; we will not stop supporting it and following its developments hoping that the commitment of each one of us can bring the desired results.

Together we are stronger.

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