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Avidity Therapeutics
San Diego, California, United States
- Ownership
- Private
- Employees
- ~120
- Development stage
- Other
01
Overview
Avidity has advanced three RNA therapeutic programs into pivotal or late-stage clinical development for rare neuromuscular diseases. Their AOC 1001 program (delpacibart etedesiran, 'del-desiran') for myotonic dystrophy type 1 is in a global Phase 3 trial (HARBOR™), showing successful muscle RNA delivery. Phase 2 and pivotal clinical trials are ongoing for facioscapulohumeral muscular dystrophy (AOC 1020, FORTITUDE™ study) and Duchenne muscular dystrophy with exon 44 skipping (AOC 1044, EXPLORE44-OLE™ study). Clinical results highlight unprecedented targeted delivery, molecular response, and early signals of clinical benefit.[2][3][4][5][6]
- Therapeutic areas
- NeurologyRare Diseases
- Modalities
- Antibody-oligonucleotide conjugateOligonucleotideSmall interfering RNAPhosphorodiamidate morpholino oligomer
- Industry
- Biotech
02
Partnerships
Bristol Myers Squibb (precision cardiology collaboration, up to five targets)Eli Lilly (immunology collaboration)
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