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

*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.