SAN DIEGO, Calif., February 26, 2026
Avidity Biosciences, Inc. (Nasdaq: RNA) announced that it will present one oral and six poster presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 8–11, 2026, in Orlando, Florida. The presentations will highlight new clinical and translational data from Avidity’s proprietary Antibody Oligonucleotide Conjugate (AOC™) platform, including updated findings from its Phase 1/2 EXPLORE44 trial in Duchenne muscular dystrophy (DMD44) and ongoing programs in myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). The breadth of data underscores the company’s strategy to position AOCs as a transformative modality in RNA-targeted precision therapeutics for rare neuromuscular diseases.
One-Year Del-Zota Data Show Functional Gains
The featured oral presentation will report one-year data for delpacibart zotadirsen (del-zota; AOC 1044) in individuals living with DMD44. Results from the EXPLORE44 program demonstrate near normalization of creatine kinase (CK) levels, a key biomarker of muscle damage, alongside improvements in functional outcomes after one year of treatment. CK reduction is widely recognized as an indicator of decreased muscle breakdown, and sustained normalization suggests meaningful biological activity. In DMD, where progressive muscle degeneration leads to significant morbidity and reduced life expectancy, the ability to demonstrate both biomarker improvement and functional benefit represents a critical milestone. These findings also support the advancement of SAFARI44, a global Phase 3 trial evaluating the efficacy and safety of del-zota in DMD44, reflecting regulatory and developmental progression toward late-stage evaluation.
Expanding Pipeline in DM1 and FSHD
In addition to DMD44 updates, Avidity will present data across its broader neuromuscular pipeline. Poster sessions will include patient and caregiver experience research in myotonic dystrophy type 1 (DM1), data from participants treated with del-desiran in the MARINA-OLE extension study, and mechanistic insights into DUX4-driven gene regulation in FSHD cell line models. Additional analyses from the EXPLORE44 trial will detail RNA sequencing (RNASeq) findings demonstrating improvements in inflammatory and fibrotic disease signatures in DMD muscle tissue, reinforcing the molecular impact of the AOC platform. Collectively, these presentations emphasize the integration of clinical endpoints, biomarker validation, and patient-centered outcomes in Avidity’s development framework.
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The company will also host an industry forum during the conference focused on biomarker advancements in rare neuromuscular diseases, highlighting the growing importance of translational science and data-driven endpoints in regulatory evaluation and long-term therapeutic positioning.
AOC Platform Driving Targeted RNA Delivery
Avidity’s AOC™ platform is designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, enabling targeted delivery of RNA therapeutics to muscle tissue. The company has previously reported the first successful targeted delivery of RNA into muscle, addressing a long-standing challenge in RNA drug development. Beyond neuromuscular disorders, Avidity is advancing precision cardiology programs targeting rare genetic cardiomyopathies, while expanding partnerships in cardiology and immunology.
As regulatory agencies increasingly prioritize validated biomarkers, functional endpoints, and patient-reported outcomes in rare disease drug development, Avidity’s integrated approach positions its RNA therapeutics pipeline at the forefront of innovation. The comprehensive data set to be presented at MDA 2026 reinforces the company’s commitment to advancing precision RNA medicines with scalable clinical and commercial potential.
Source: Avidity Biosciences, Inc. press release
