| 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 |
*Sept. 2022, FDA placed a partial clinical hold on new participant enrollment. All current participants may continue in their current dosing cohort. All participants in MARINA may roll over into the MARINA-OLE where they will receive AOC 1001 as planned.
Avidity is working to resolve the partial clinical hold as quickly as possible.