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Avidity Biosciences Inc (RNA)
NASDAQ:RNA
Aoc-1020
Facioscapulohumeral Muscular Dystrophy 1, Fshd, Fshd1, Fshd2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Fsh Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Landouzy-Dejerine Syndrome, Facioscapulohumeral Muscular Dystrophy, Fshd - Facioscapulohumeral Muscular Dystrophy, Facioscapulohumeral Dystrophy, Facioscapulohumeral Muscular Dystrophy Type 1 (Fshd1)
Phase III
Recruiting
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
Jun 13, 2025
Aoc 1020
Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy 1, Fshd, Fshd1, Fshd2, Fmd, Fmd2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophy, Facioscapulohumeral, Fsh Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, Fsh
Phase II
Enrolling By Invitation
Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Aug 07, 2024
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Aoc 1001 (Del-Desiran)
Dm1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (Dm1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy, Myotonia, Steinert
Phase III
Active Not Recruiting
Global Study of Del-desiran for the Treatment of DM1
May 08, 2024
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Aoc 1044
Duchenne Muscular Dystrophy, Dmd, Duchenne, Exon 44
Phase II
Active Not Recruiting
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
Jan 26, 2024
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Aoc 1020
Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy 1, Fshd, Fshd1, Fshd2, Fmd, Fmd2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophy, Facioscapulohumeral, Fsh Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, Fsh
Phase I/II
Active Not Recruiting
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Feb 17, 2023
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Aoc 1044
Duchenne Muscular Dystrophy, Exon 44
Phase I/II
Completed
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
Dec 06, 2022
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Aoc 1001
Nervous System Diseases, Neurodegenerative Diseases, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Genetic Diseases, Inborn, Dm1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Disorders, Heredodegenerative Disorders, Nervous System
Phase II
Active Not Recruiting
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Jul 27, 2022
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Aoc 1001
Dm1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (Dm1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
Phase I/II
Completed
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Aug 24, 2021
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FAQ
What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases:
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does Avidity Biosciences Inc (RNA) have in its pipeline
RNA is currently developing the following drugs: Aoc-1020, Del-Desiran (Aoc 1001), Aoc 1020. These drug candidates are in various stages of clinical development as the company works toward FDA approval.