Pipeline | Overview

Delivering on the RNA Revolution

We are advancing and expanding our innovative AOC pipeline to offer treatment options for patients and their families across a wide range of therapeutic areas. We have three AOC programs for three distinct rare disease in clinical development from our muscle disease franchise: myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne Muscular Dystrophy (DMD). We are also advancing AOCs for additional DMD programs, rare skeletal muscle, rare cardiac program, and continue to broaden our reach of AOCs to other indications like cardiology and immunology through internal discovery & partnerships.

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

*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.