Program/Indication | Target | Lead Optimization | IND Enabling | Phase 1/2 | Phase 3 |
---|---|---|---|---|---|
AOC 1001* Myotonic Dystrophy Type 1 (DM1) | DMPK | ||||
AOC 1044 Duchenne Muscular Dystrophy (DMD) | Exon 44 | ||||
AOC 1020 Facioscapulohumeral Muscular Dystrophy (FSHD) | DUX4 | ||||
Additional DMD Programs | Exon 45 & Undisclosed | ||||
Rare Skeletal Muscle Program | Undisclosed | ||||
Rare Cardiac Program | Undisclosed |
Program/Indication | Target | Stage |
---|---|---|
AOC 1001* Myotonic Dystrophy Type 1 (DM1) | DMPK | Phase 1/2 |
AOC 1044 Duchenne Muscular Dystrophy (DMD) | Exon 44 | Phase 1/2 |
AOC 1020 Facioscapulohumeral Muscular Dystrophy (FSHD) | DUX4 | Phase 1/2 |
Additional DMD Programs | Exon 45 & Undisclosed | IND Enabling |
Rare Skeletal Muscle Program | Undisclosed | IND Enabling |
Rare Cardiac Program | Undisclosed | IND Enabling |
*In May 2023, the FDA eased the partial clinical hold placed in September 2022 to allow a number of current participants to be dose escalated to 4 mg/kg of AOC 1001 and new participant enrollment at 2 mg/kg of AOC 1001.