Program/IndicationTargetDiscovery/Lead OptimizationIND EnablingPhase 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/IndicationTargetStage
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