Update on DMD gene therapy trial

Published Date
Sofia Nnorom
Orange box stating: Breaking news research

Sarepta Therapeutics has released an update on the four boys dosed in its ongoing gene therapy trial. The data was presented by Dr Jerry Mendell, the trial’s principal investigator, at the World Muscle Society conference yesterday.

Building on Sarepta’s previous data, Dr Mendell shared results on the fourth boy treated with the gene therapy (AAVrh74.MHCK7.microdystrophin). Like the other participants, he had a significant increase in the dystrophin protein and a reduction in creatine kinase levels – a marker for muscle damage. So far, there have been no serious safety concerns reported for any of the participants.

Dr Mendell also reported that all four boys have shown improvements in functional assessments (e.g. stair climbing, standing up and walking ability) compared to the start of the study. Whilst these preliminary results are very exciting and promising, it should be cautioned that this study is very small and has no controls.

Sarepta is now planning to conduct a larger trial in the US – data from this trial will be used to seek marketing approval for the therapy. The company is mapping ways to expand its clinical program and will announce an update once it finalises its plans.

In a press release, Doug Ingram, Sarepta’s president and chief executive officer said:

The encouraging results that we previously saw and reinforced in the fourth patient strengthen our resolve to rapidly move to a confirming trial and, assuming successful, to bring this therapy to the Duchenne community around the world with a sense of urgency.

Please read Sarepta’s community letter or press release for further information.

If you have any questions about this news story or any other Duchenne research, please contact the MDUK Research Line on 020 7803 4813 or email research@musculardystrophyuk.org

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