Avidity Biosciences
Avidity Biosciences works on 3 rare diseases tracked on Trial Friend, including Duchenne Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy, with 5 active clinical trials (3 recruiting).
Avidity Biosciences is a San Diego biotech built around a delivery problem that stumped the field for decades: RNA medicines are powerful, but they could not reach muscle tissue. Avidity's answer is the antibody oligonucleotide conjugate (AOC), which attaches an RNA drug to a monoclonal antibody that homes to a receptor on muscle cells, carrying the payload where naked RNA cannot go.
That platform produced three late-stage programs in three different muscular dystrophies, and Novartis bought the company to get them. The deal was announced in October 2025 and completed on February 27, 2026, at $72.00 per share in cash, valuing Avidity at approximately $12 billion. Avidity is now a wholly owned Novartis subsidiary and no longer trades publicly.
The three programs have gone in different directions since. Del-desiran, for myotonic dystrophy type 1, did not meet the primary endpoint of its Phase 3 HARBOR study. Novartis reported on September 8, 2026 that the drug did not significantly improve video hand opening time, a measure of hand myotonia, compared with placebo. Novartis said there was evidence of activity on some secondary and exploratory measures, that safety was consistent with earlier data, and that it is reviewing the full dataset with regulators to determine whether and how the program continues. Del-zota, for Duchenne muscular dystrophy in patients amenable to exon 44 skipping, has been filed for accelerated approval and granted FDA priority review. Del-brax, for facioscapulohumeral muscular dystrophy (FSHD), met its primary endpoint in a Phase 1/2 study, and Novartis plans to meet with the FDA on next steps based on that biomarker data.
For patients, the picture is mixed. Two of the three programs are moving toward regulators with the scale of a large company behind them. Myotonic dystrophy type 1 still has no approved treatment aimed at its genetic cause, and the HARBOR result means that remains true for now.
Avidity Biosciences Drug Pipeline
Avidity Biosciences has 5 active clinical trials across 3 development stages, with 3 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Avidity Biosciences Clinical Trials (5)
Active and recruiting clinical trials sponsored by Avidity Biosciences, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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FDA-Approved Drugs
Avidity Biosciences Trial Locations
Avidity Biosciences clinical trials are running at 112 sites in 13 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (3)
Diseases targeted by Avidity Biosciences's clinical trial and drug development programs
Duchenne muscular dystrophy is an X-linked genetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood. The defective dystrophin protein normally protects muscl...
Facioscapulohumeral Muscular Dystrophy (FSHD) is an inherited muscular dystrophy characterized by progressive weakness of the facial, shoulder, and upper arm muscles. The condition results from abnorm...
Myotonic Dystrophy (MD) is the most common muscular dystrophy in adults, characterized by progressive muscle weakness and myotonia—the inability of muscles to relax after contraction. The condition re...
Patient Resources
Organizations and resources related to Avidity Biosciences's rare disease focus areas
Frequently Asked Questions About Avidity Biosciences
Common questions about Avidity Biosciences's rare disease programs, clinical trials, and treatments.