International gene therapy trial starts in France 

Published Date
MDUK Research
Scientist observing a DNA molecule

We’re delighted to tell you that the first boy with Duchenne muscular dystrophy has been dosed as part of a clinical trial to study a gene therapy for the condition.

The investigational gene therapy, called GNT 0004, is part of an international phase I/II/III multicentre clinical trial sponsored by Genethon. The boy was at I-Motion, the paediatric clinical trial platform for neuromuscular diseases in Paris.

The trial will assess the safety and efficacy of GNT 0004. It uses an AAV- type viral vector to deliver microdystrophin, a shortened version of the dystrophin gene, to the cells of the body. Genethon developed GNT 0004 in partnership with Prof George Dickson and his team at Royal Holloway, University of London, with part funding from MDUK – thanks to the generosity of our donors who’ve supported this research over the years.

Prof Francesco Muntoni from the Dubowitz Neuromuscular Centre at UCL Great Ormond Street Institute of Child Health is the Global Chief Investigator for the trial. He tells us:

Individuals with Duchenne muscular dystrophy remain in need of effective treatments. This is a fantastic first step for this clinical trial to investigate this gene therapy. We aim to start including boys from the UK in the trial before the end of 2021.

You can read more about some of the background to the Genethon trial here.

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