forazapadin

Forazapadin (formerly SAT-3247) is an orally administered, small molecule inhibitor of AP2-associated protein kinase 1 (AAK1) being developed by Satellos Bioscience. It is designed as a novel, dystrophin-independent regenerative therapy to restore natural muscle repair and regeneration in degenerative muscle diseases. In Duchenne muscular dystrophy (DMD), the absence of dystrophin disrupts asymmetric division of muscle stem cells, impairing muscle repair. By targeting and inhibiting AAK1, forazapadin aims to re-establish the biochemical signals required for muscle stem cells to divide and differentiate into new muscle tissue. The drug is currently in Phase 2 clinical development for Duchenne muscular dystrophy (DMD) and is also being evaluated for facioscapulohumeral muscular dystrophy (FSHD).

Other names

forazapadinum