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 diseases in clinical development from our muscle disease franchise: myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne Muscular Dystrophy (DMD). Our pipeline also includes advancing AOCs to address additional DMD, rare skeletal muscle, and rare precision cardiology programs. We continue to broaden our reach of AOCs in other indications including cardiology and immunology through partnerships.

Program/Indication	Target	Lead Optimization	IND Enabling	Phase 1/2	Phase 3

Del-desiran (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
Del-desiran (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

^*Phase 3 HARBOR Trial Initiation planned for Q224