Pipeline Overview

Delivering on the RNA Revolution

We are advancing and expanding our innovative AOC pipeline to offer treatment options for patients and their families across a wide range of therapeutic areas. We have three AOC programs for three distinct rare diseases in clinical development from our muscle disease franchise: myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne Muscular Dystrophy (DMD). Our pipeline also includes advancing AOCs to address additional DMD and rare neuromuscular programs.

Study drug(s) and AOC™ technology are investigational. Efficacy and safety have not been established and there is no guarantee that the therapies will become commercially available for the use(s) under investigation.