SMT C1100

Increasing the protein utrophin to compensate for missing dystropin
Scientists have shown that increasing the production of the protein utrophin in Duchenne patients can compensate for the missing dystrophin, and help restore healthy muscle function. The Duchenne Research Fund was involved in funding some of the pre-clinical development of a small molecule utrophin modulator for Duchenne, developed by Summit Therapeutics. The research was spearheaded by Professor Dame Kay Davies and her group at Oxford University. This treatment was intended to complement other therapies and to benefit all boys regardless of type of mutation.

SMT C1100 was tested in early stage clinical trials, where it was shown to be safe. A further Phase 1b trial in patients with Duchenne finished in 2015 and achieved its primary objective, enabling it to progress to Phase 2 clinical trials.

Professor Davies and her team went on to work in collaboration with Professor Angela Russell as part of the UtroDMD Alliance to identify second- and future-generation compounds that are better able to increase utrophin levels and have better absorption and distribution profiles in the body, which would make them better candidates for a drug.

Pre-clinical research

Funded in 2010

Oxford, UK

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