2003-2007

Genethon IV – Leading the Way in Human Trials

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The advances made in understanding rare genetic disorders and in the development of gene therapy vectors led Genethon to set up a number of preclinical programs. While continuing with its extensive research activities, Genethon put in place the various structures that would be needed to start clinical trials in humans.

The year 2004 saw another World First for Genethon, when Olivier Danos and Luis Garcia succeeded in restoring dystrophin expression to the limb muscles in a murine model of Duchenne muscular dystrophy using exon skipping (journal Science, December 3, 2004).

In 2005, its GMP-compliant vector production site was certified by the French Healthcare Products Safety Agency (AFSSAPS), and became a Gene and Cell Therapy Facility (as per French law).

In 2006, Genethon IV was authorized to launch its first gene therapy trial for gamma-sarcoglycanopathy. At the same time, clinical grade vector preparations were underway for two other trials in Duchenne muscular dystrophy and Wiskott-Aldrich syndrome.