Program/IndicationTargetLead OptimizationIND EnablingPhase 1/2Phase 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/IndicationTargetStage
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.