We were delighted to hear the recent news that Sarepta Therapeutics has partnered with Genethon. This has positive implications for the UNITE-DMD project that we are co-funding with Action Duchenne and the French Muscular Dystrophy Association (AFM).
UNITE-DMD builds on the collaborative work of Professor George Dickson from Royal Holloway University and Genethon. Over the last decade, they have designed and refined a micro-dystrophin gene therapy for Duchenne muscular dystrophy. This research has been largely supported by MDUK and AFM.
This micro-dystrophin gene therapy will be tested in a phase I/II clinical trial as part of the four-year UNITE-DMD project. Although Sarepta is not directly involved with UNITE-DMD, its partnership with Genethon is extremely beneficial and strengthens the project’s prospects. It will contribute to the necessary financial resources and commercial expertise to manage future clinical trials and regulatory approval processes.
Professor George Dickson, one of the leaders of UNITE-DMD, said:
Sarepta and Genethon working together, along with patient-centred organisations like MDUK and AFM, is a powerful combination. It will help us to achieve the ultimate goal of bringing a safe and effective DMD gene therapy into routine clinical use.
Dr Jenny Versnel, Director of Research and Business Innovation at MDUK, said:
This partnership between Sarepta and Genethon is very exciting and will help to advance the UNITE-DMD project in future. The collaborative input of three patient organisations into this project will ensure that the voice of the Duchenne community is heard.
Diana Ribeiro, CEO of Action Duchenne, said:
Collaboration is vital to the Duchenne community. We are excited to see the benefits this partnership between Sarepta and Genethon, can bring to the UNITE-DMD project and what it will mean for developing the gene therapy approach for the global community.
Find out more about our strategic investments, including UNITE-DMD
Learn more about AAV gene therapy for muscle-wasting conditions
Find out more on MDUK-funded Duchenne research
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