SAT-3247 is an oral small molecule inhibitor of AAK. SAT-3247 seeks to restore asymetric divsion of stem cells in muscle, which is disrupted by the lack of dystrophin.By helping to restore asymetric division SAT-3247 may help to allow for stem cells to differentiate into new muscle tissue and thereby increase muscle regeneration and provide benefit to individuals with Duchenne.
A phase 1 study in healthy volunteers and adults with DMD is recruiting in Australia.
This program is sponsored by Satellos Bioscence and has been partially funded by Parent Project Muscular Dystrophy.
ACTIVELY RECRUITING | First in Human SAD/MAD Safety and PK Study with Adult DMD Safety and PK Cohort |
