| Program/Indication | Target | Discovery/Lead Optimization | IND Enabling | Phase 1/2 |
|---|---|---|---|---|
| AOC 1001* Myotonic Dystrophy Type 1 (DM1) | DMPK | |||
| AOC 1020 Facioscapulohumeral Muscular Dystrophy (FSHD) | DUX4 | |||
| AOC 1044 Duchenne Muscular Dystrophy (DMD) | Exon 44 Dystrophin | |||
| Next AOC DMD Programs | Exon 51 Dystrophin | |||
| Exon 45 Dystrophin | ||||
| AOC Muscle Atrophy Muscle Atrophy** | MuRF1 | |||
| AOC Pompe Disease Pompe Disease | GYS1 |
| Program/Indication | Target | Stage |
|---|---|---|
| AOC 1001* Myotonic Dystrophy Type 1 (DM1) | DMPK | Phase 1/2 |
| AOC 1020 Facioscapulohumeral Muscular Dystrophy (FSHD) | DUX4 | Phase 1/2 |
| AOC 1044 Duchenne Muscular Dystrophy (DMD) | Exon 44 Dystrophin | Phase 1/2 |
| Next AOC DMD Programs | Exon 51 Dystrophin | Discovery/Lead Optimization |
| Exon 45 Dystrophin | Discovery/Lead Optimization | |
| AOC Muscle Atrophy Muscle Atrophy** | MuRF1 | Discovery/Lead Optimization |
| AOC Pompe Disease Pompe Disease | GYS1 | Discovery/Lead Optimization |
*Sept. 2022, U.S. Food and Drug Administration (FDA) placed a partial clinical hold on new participant enrollment. All current participants may continue in their current dosing cohort. Avidity is working closely with the FDA and the trial investigator to resolve the partial clinical hold as quickly as possible.
**Opportunity for a rare disease indication