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Green light for a Duchenne muscular dystrophy gene therapy in France

7 December 2020

These are exciting times for the development of treatments, some of which have the potential to markedly improve outcomes for a number of neuromuscular conditions.

We recently posted a blog from Rob Burley, MDUK’s Director for Campaigns, Care and Support, on a number of potential treatments currently being appraised.

To add to that excitement, Genethon announced last week that they had received authorisation from the French National Agency for Medicines and Health Products Safety to start the French arm of a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy, with product GNT 004. This means that there are now four gene therapies for Duchenne muscular dystrophy being trialled internationally.

The Genethon study is an evolution of the Universal Microdystrophin (MD) Gene Therapy Clinical Trial for DMD (UNITE-DMD) project. Thanks to support from our community, MDUK has contributed in the region of £440k towards the UNITE-DMD study.

For over 20 years, we’ve supported research to design, test and refine a microdystrophin AAV gene therapy for Duchenne muscular dystrophy. MDUK funding has always been at the core of this effort, having funded Prof George Dickson at Royal Holloway University of London for his work on exon skipping and gene therapy, along with the French charity AFM.

In addition to supporting the work at Royal Holloway, we have supported Prof Francesco Muntoni, Dr Adnan Manzur and their teams to establish the NorthStar Neuromuscular Database, which has been world-leading in collecting natural history data on boys with Duchenne muscular dystrophy for over a decade.

NorthStar data provides immense power because its clinical scale is the primary outcome measure for many clinical trials. Data from the ongoing natural history study, which links all paediatric UK centres, can also inform the design of clinical trials, including the one using the product GNT 004.

Until recently, MDUK was funding the clinical work in the UK of the UNITE-DMD study. We’re delighted that Genethon and their partners, Sarepta, have taken over the financial support for this work and are working with clinical research teams in France, in the UK (at UCL and Newcastle) and at other international sites to take the trial forward. We anticipate the UK arm of the study will start in the next few months.

MDUK continues to support a programme of work at Royal Holloway, led by Dr Linda Popplewell, to look at further refinement of microdystrophin and the AAV viral vector that is used to deliver the gene to cells.

While we’re excited by these developments, as ever, we’re mindful that the work is still at the early phase of clinical trials. There is still a lot of safety and efficacy testing to be done, so it’s likely to be several years before commercialisation discussions take place. The Genethon/Sarepta partnership means that there is a pathway to making the gene therapy available to patients (a process called commercialisation). Sarepta will work with NICE and the NHS to find a way to make the treatment available in the UK.

Dr Kate Adcock, Director of Research and Innovation at MDUK says:

“We are proud that as a medium-sized charity based in the UK, MDUK has been able to contribute to the future delivery of the microdystrophin gene therapy product generated by Genethon in collaboration with the Royal Holloway lab. The UNITE-DMD programme has evolved to such a place that it makes sense for the clinical work to be fully supported by the sponsors, Genethon.”

Prof Francesco Muntoni, Director of the Dubowitz Neuromuscular Centre at Great Ormond Street Hospital and Global Chief Investigator for the study, says:

“This clinical trial would not have been possible without MDUK’s vision and long-term support of translational research and natural history studies in the UK. This research underpins much of the early development of this clinical trial.”

Prof Volker Straub, Director of the John Walton Muscular Dystrophy Research Centre at Newcastle University says:

“It is excellent news for the DMD community that another gene therapy trial has launched. We anticipate starting the natural history study in the UK in the coming months, following ethical approval, and are looking forward to the new interventional gene therapy trial.”

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