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. Our first AOC programs are from our muscle disease franchise which includes programs in myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular Dystrophy (DMD), muscle atrophy and Pompe disease. We are also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key 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

*Sept. 2022, FDA placed a partial clinical hold on new participant enrollment. All current participants may continue in their current dosing cohort. Avidity is working to resolve the partial clinical hold as quickly as possible.