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Avidity Biosciences, Inc. (Nasdaq: RNA) is a biopharmaceutical company focused on developing a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs™). According to the company, its mission is to profoundly improve people's lives by delivering AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases that have been difficult to reach with existing RNA therapies. Avidity is headquartered in San Diego, California and operates in the pharmaceutical preparation manufacturing segment of the broader manufacturing sector.

Avidity states that it has demonstrated the first-ever successful targeted delivery of RNA into muscle using its proprietary AOC platform. Building on this platform, the company is advancing multiple clinical development programs in rare neuromuscular diseases, specifically myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). These diseases are characterized by progressive muscle weakness and significant functional impairment, and in some cases, a lack of approved treatments. Avidity’s work is positioned around addressing the high unmet medical need in these indications.

Core AOC Platform and Therapeutic Focus

The company describes its AOCs as molecules that bring together monoclonal antibodies and oligonucleotide therapies. The monoclonal antibody component is designed to provide target specificity, while the oligonucleotide component is intended to modulate RNA inside cells. By linking these two elements, Avidity aims to deliver RNA-targeting therapies directly to tissues such as skeletal and cardiac muscle. This approach is central to its programs in rare muscle diseases and to its broader pipeline in cardiology and immunology through internal discovery and partnerships.

Avidity’s clinical-stage neuromuscular portfolio includes three named AOC programs that the company has highlighted in its public communications:

• Delpacibart zotadirsen (del-zota) for Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44).
• Delpacibart etedesiran (del-desiran) for myotonic dystrophy type 1 (DM1).
• Delpacibart braxlosiran (del-brax) for facioscapulohumeral muscular dystrophy (FSHD).

According to company disclosures, all three programs are in late-stage clinical development. Avidity reports that it has generated what it describes as groundbreaking or unprecedented data across these neuromuscular programs, including reversal of disease progression and improvements across multiple functional measures in people living with DMD44 treated with del-zota in the EXPLORE44® and EXPLORE44-OLE™ trials. The company also notes that its DM1 program has advanced into a global Phase 3 study (HARBOR™), and that it has initiated a global, confirmatory Phase 3 study (FORTITUDE-3™) intended to support a global approval strategy for del-brax in FSHD.

Lead Program: Del-zota in Duchenne Muscular Dystrophy (DMD44)

Duchenne muscular dystrophy is described by Avidity as a monogenic, X-linked, recessive disease that primarily affects males and is characterized by a lack of functional dystrophin. This lack of dystrophin leads to stress and tears of muscle cell membranes, muscle cell death, and progressive loss of muscle function. People living with DMD typically experience early-onset progressive muscle weakness, eventually developing problems walking and breathing, with heart and respiratory muscles ultimately affected. While there are approved treatments for DMD, the company notes that there remains a very high unmet need, and that there are no approved exon 44 skipping therapies for DMD44.

Del-zota is described as an investigational AOC designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene, with the goal of enabling dystrophin production in people with DMD44. The molecule consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 (TfR1) conjugated to a PMO targeting exon 44. In the Phase 1/2 EXPLORE44 trial and the EXPLORE44-OLE™ extension, Avidity reports that del-zota achieved what it characterizes as unsurpassed delivery of PMOs to skeletal muscle, robust increases in dystrophin production, significant increases in exon 44 skipping, and significant and sustained decreases of creatine kinase levels to near normal.

Functional data disclosed by the company indicate that participants treated with del-zota showed improvements compared to DMD natural history across several measures, including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and the 10-Meter Walk/Run Test (10mWRT). Avidity also reports that del-zota continued to demonstrate a favorable long-term safety and tolerability profile, with most treatment-emergent adverse events described as mild or moderate, and one discontinuation due to hypersensitivity.

The company has announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to del-zota for DMD44. Following a pre-Biologics License Application (BLA) meeting, Avidity has stated that it aligned with the FDA on a path forward for a BLA submission for del-zota, with the timing updated to the first quarter of 2026 to provide additional data to support the chemistry, manufacturing, and controls package. The company has also described plans for a confirmatory study to support full global approval of del-zota.

Del-desiran in Myotonic Dystrophy Type 1 (DM1)

Myotonic dystrophy type 1 is described as an underrecognized, progressive and often fatal disease caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain-of-function mRNA. Avidity notes that DM1 is highly variable in severity, presentation and age of onset, but that all forms are associated with high disease burden and may cause premature mortality. The disease primarily affects skeletal and cardiac muscle, and can involve myotonia, muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, gastrointestinal complications, and cognitive and behavioral impairment. The company states that there are no approved treatments for people living with DM1.

Del-desiran is Avidity’s AOC candidate for DM1. The company reports that it has advanced del-desiran into the global Phase 3 HARBOR™ trial, which it describes as the first global Phase 3 trial of this agent for DM1. Avidity has also referenced earlier-stage data from the Phase 1/2 MARINA® trial and has indicated plans for data analyses and readouts from HARBOR™. These disclosures position del-desiran as a key part of Avidity’s late-stage neuromuscular pipeline.

Del-brax in Facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral muscular dystrophy is described as a rare, progressive, and variable hereditary muscle-weakening condition marked by pain, fatigue, disability and progressive skeletal muscle loss. It typically begins with weakness in muscles of the face, shoulders, arms and trunk, and can progress to the lower body, often leading to wheelchair dependence. FSHD is attributed to aberrant expression of the DUX4 gene in skeletal muscle, which activates genes that are toxic to muscle cells and leads to muscle wasting and compromised function. Avidity notes that there are currently no approved treatments for people living with FSHD.

Del-brax is Avidity’s AOC candidate for FSHD. The company has described topline data expectations from the FORTITUDE™ biomarker cohort and has indicated that it has aligned with the FDA on accelerated and full approval pathways for del-brax. Avidity has also reported initiation of the global, confirmatory Phase 3 FORTITUDE-3™ study, which is intended to support a global approval strategy for del-brax.

Broader Pipeline: Cardiology and Immunology

Beyond neuromuscular diseases, Avidity reports that it is advancing two wholly owned precision cardiology development candidates that address rare genetic cardiomyopathies. The company has identified programs targeting phospholamban (PLN) and Protein Kinase AMP-activated non-catalytic subunit Gamma 2 (PRKAG2) syndrome as part of its early-stage precision cardiology efforts, which are expected to be separated into a new public company (SpinCo) in connection with the proposed acquisition by Novartis AG.

Avidity also states that it is broadening the reach of AOCs with an advancing and expanding pipeline that includes programs in cardiology and immunology through key partnerships. The company has disclosed collaboration and license agreements with partners such as Eli Lilly and Company and Bristol Myers Squibb, and has reported collaboration revenues and milestones associated with these arrangements.

Planned Acquisition by Novartis and Spin-Off of Precision Cardiology Programs

Avidity has entered into an Agreement and Plan of Merger with Novartis AG and an indirect wholly owned Novartis subsidiary. Under this agreement, the subsidiary is expected to merge with and into Avidity, with Avidity surviving as an indirect wholly owned subsidiary of Novartis, subject to customary closing conditions. In connection with the merger, Avidity has also entered into a Separation and Distribution Agreement providing for a pre-closing reorganization in which a newly formed subsidiary (SpinCo) will own assets and liabilities related to early-stage precision cardiology programs and certain collaboration agreements.

Following this reorganization, Avidity has disclosed that SpinCo is expected either to be distributed to Avidity stockholders on a pro rata basis (the Spin-Off Distribution) or to be sold to a third party, subject to the terms of the merger agreements. Novartis is expected to acquire Avidity’s programs and pipeline in neuroscience and to gain access to its RNA-targeting delivery platform, including the late-stage neuromuscular programs del-zota, del-desiran and del-brax. The companies have stated that they expect the transactions to close in the first half of 2026, subject to completion of the SpinCo separation, regulatory approvals, stockholder approval and other customary conditions.

Regulatory filings indicate that the Federal Trade Commission granted early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act for the transactions, satisfying one of the conditions to closing. Other conditions, including completion of the SpinCo separation and stockholder approval, remain to be satisfied or waived before the merger can be consummated.

Advocacy Engagement and Patient Community Focus

Avidity emphasizes engagement with patient and advocacy communities, particularly in the context of neuromuscular and rare diseases. During National Muscular Dystrophy Awareness Month, the company has described activities such as supporting World Duchenne Awareness Day, International Myotonic Dystrophy Awareness Day, the FSHD Society Walk & Roll to Cure FSHD, and Global Genes Week in RARE. It has also referenced participation in events organized by groups such as the Jett Foundation and Defeat Duchenne Canada, reflecting an emphasis on collaboration with patient organizations and awareness initiatives.

Stock Listing and Regulatory Filings

Avidity’s common stock, with a par value of $0.0001 per share, is listed on The Nasdaq Global Market under the trading symbol RNA. The company files periodic and current reports, including Forms 8-K, with the U.S. Securities and Exchange Commission (SEC). These filings cover topics such as clinical data updates, regulatory designations, public offerings of common stock, collaboration milestones, and details of the proposed merger and SpinCo separation. Investors and analysts use these filings to track material events, transaction terms, and developments in Avidity’s clinical and corporate strategy.

FAQs about Avidity Biosciences, Inc. (RNA)

• What does Avidity Biosciences do?
  Avidity Biosciences is a biopharmaceutical company developing Antibody Oligonucleotide Conjugates (AOCs™), a class of RNA therapeutics that combine monoclonal antibodies with oligonucleotide therapies. The company focuses on rare neuromuscular diseases such as myotonic dystrophy type 1, Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy, and is also advancing precision cardiology and other programs through internal efforts and partnerships.
• What is an Antibody Oligonucleotide Conjugate (AOC)?
  According to Avidity, an AOC is a molecule that links a monoclonal antibody to an oligonucleotide therapy. The antibody component is designed to provide target specificity, while the oligonucleotide component modulates RNA inside cells. This design is intended to enable targeted delivery of RNA therapeutics to tissues such as muscle, addressing diseases that have been difficult to treat with existing RNA approaches.
• Which diseases are the focus of Avidity’s lead clinical programs?
  Avidity’s lead clinical programs target three rare muscle diseases: myotonic dystrophy type 1 (DM1) with del-desiran, Duchenne muscular dystrophy (DMD), including mutations amenable to exon 44 skipping (DMD44) with del-zota, and facioscapulohumeral muscular dystrophy (FSHD) with del-brax. The company has described these programs as late-stage clinical development efforts.
• What is del-zota and who might it help?
  Delpacibart zotadirsen (del-zota) is an investigational AOC designed for people living with Duchenne muscular dystrophy who have gene mutations amenable to exon 44 skipping (DMD44). It links a monoclonal antibody targeting transferrin receptor 1 to a phosphorodiamidate morpholino oligomer that targets exon 44 of the dystrophin gene. Avidity reports that del-zota has shown increases in dystrophin production, exon skipping, and improvements in functional measures in clinical trials, and that it has received Breakthrough Therapy designation from the FDA.
• What regulatory milestones has del-zota achieved?
  Avidity has disclosed that the U.S. Food and Drug Administration granted Breakthrough Therapy designation to del-zota for DMD44. Following a pre-BLA meeting, the company reported alignment with the FDA on a path forward for a Biologics License Application submission and updated its expected timing for submission to the first quarter of 2026 to provide additional chemistry, manufacturing and controls data.
• What is the HARBOR™ trial in DM1?
  The HARBOR™ trial is described by Avidity as a global Phase 3 study of del-desiran in people living with myotonic dystrophy type 1. The company has announced completion of enrollment for this trial and has indicated plans for topline data readouts, positioning HARBOR™ as a key step toward potential regulatory submissions in DM1.
• How is Avidity involved in FSHD research?
  Avidity is developing del-brax for facioscapulohumeral muscular dystrophy, a hereditary muscle-weakening condition linked to aberrant expression of the DUX4 gene. The company has reported plans for biomarker cohort data from the FORTITUDE™ program and has initiated the global Phase 3 FORTITUDE-3™ study, which is intended to support a global approval strategy for del-brax.
• What is SpinCo and how does it relate to Avidity?
  In connection with a planned acquisition by Novartis AG, Avidity has agreed to transfer assets and liabilities related to its early-stage precision cardiology programs and certain collaboration agreements into a separate entity referred to as SpinCo. SpinCo is expected either to be distributed to Avidity stockholders or sold to a third party before the merger closes. After the separation, Avidity will not retain an ownership interest in SpinCo.
• Is Avidity being acquired?
  Avidity has entered into a definitive merger agreement under which an indirect wholly owned subsidiary of Novartis AG will merge with and into Avidity, with Avidity becoming an indirect wholly owned subsidiary of Novartis. The transaction is subject to conditions including completion of the SpinCo separation, regulatory approvals and stockholder approval. Company disclosures state that the parties expect the transactions to close in the first half of 2026, but closing has not yet occurred based on the available information.
• On which exchange does Avidity trade and what is its ticker symbol?
  Avidity Biosciences’ common stock is listed on The Nasdaq Global Market under the ticker symbol RNA, as indicated in its SEC filings.