Targeting muscle fibrosis

HT-100 is a drug aimed at halting or even reversing fibrosis, the muscle scarring in Duchenne boys. It is intended to benefit all boys, regardless of type of mutation. We were part of a consortium of 12 Duchenne patient foundations that provided $1.1 million in 2012 at the start of the project, conducted by Akashi Therapeutics (formerly Halo Therapeutics).

Preliminary analyses of Phase 1 clinical trial results showed a potential muscle strength improvement, and in January 2016 Akashi received further industry funding to continue the research. The drug is currently in Phase 2 of clinical trial.

Pre-clinical research

£128,500 contribution

Funded in 2012

Cambridge, MA, USA