Avidity Biosciences joined PPMD for a community webinar on November 19, 2025, during which the company shared topline and functional data from their EXPLORE44-OLE® clinical trial. Avidity’s investigational therapy, delpacibart zotadirsen (abbreviated as del-zota), is...

Today, Avidity Biosciences announced new data from its EXPLORE44® and EXPLORE44-OLE™ Phase 1/2 studies of del-zota in people living with Duchenne muscular dystrophy amenable to exon 44 skipping. Del-zota is designed to deliver phosphorodiamidate morpholino...

Reported Wednesday, Sep 10 - 7:03am EDT (TheFly subscription required to read more.)

Reported Monday, Mar 17 - 7:09am EDT (TheFly subscription required to read more.)

Reported Monday, Mar 17 - 7:02am EDT (TheFly subscription required to read more.)

Investing.com -- Avidity Biosciences (NASDAQ:RNA) stock rose 2% after the company announced positive new data showing its experimental drug del-zota demonstrated reversal of disease progression in patients with Duchenne muscular dystrophy (DMD44). The San Diego-based biopharmaceutical company reported that patients treated continuously with del-zota for approximately one year in the EXPLORE44 and EXPLORE44-OLE trials showed "unprecedented improvement" across multiple functional measures compared to baseline and natural history data. Key functional improvements included Time to Rise from Floor (improving by 3.2 seconds), 4-Stair Climb (improving by 2.1 seconds), Performance of Upper Limb (improving by 1.5 points), and 10-Meter Walk/Run Test (improving by 0.7 seconds). In contrast, natural history comparison groups showed decline in all these measures. The company also reported a rapid reduction in creatine kinase (CK) levels to near normal levels, maintained over 16 months of follow-up, with 50% of participants having CK levels within normal range at one year. Dystrophin production increased by approximately 25% of normal. "These unprecedented data underscore the impact of our revolutionary targeted approach to deliver RNA directly to muscle," said Sarah Boyce, President and CEO of Avidity, in the press release. Del-zota is designed to deliver phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to enable production of functional, near-full length dystrophin in patients with DMD44, a rare genetic condition characterized by progressive muscle damage. The drug demonstrated a favorable long-term safety profile, with most adverse events being mild or moderate. Avidity remains on track to submit a Biologics License Application to the FDA for accelerated approval by the end of 2025. 3rd party Ad. Not an offer or recommendation by Investing.com. See disclosure here or remove ads. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C. With RNA making headlines, savvy investors are asking: Is it truly valued fairly? In a market full of overpriced darlings, identifying true value can be challenging. InvestingPro's advanced AI algorithms have analyzed RNA alongside thousands of other stocks to uncover hidden gems. These undervalued stocks, potentially including RNA, could offer substantial returns as the market corrects. In 2025 alone, our AI identified several undervalued stocks that later surged by 50% or more. Is RNA poised for similar growth? Don't miss the opportunity to find out.

Avidity Biosciences showcases promising topline data from its Phase 1/2 EXPLORE44 trial of del-zota, a potential treatment for Duchenne muscular dystrophy, demonstrating significant improvements.

Avidity Biosciences (Nasdaq: RNA) announced a U.S. Managed Access Program (MAP) to provide investigational delpacibart zotadirsen (del-zota) to eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) under an FDA-authorized treatment protocol.Enrollment is anticipated to begin by year-end 2025, and participants in EXPLORE44-OLE may transition to the MAP after completing two years of treatment. Avidity aligned with FDA after an October 2025 pre-BLA meeting and plans a BLA submission in 2026 seeking accelerated approval; patients would move to commercial supply if approved. Full MAP eligibility details will be posted on clinicaltrials.gov.

SAN DIEGO, Oct. 6, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™) to profoundly improve people's lives, today announced that it will...

-- Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and 10-Meter Walk/Run Test (10mWRT) at approximately one year -- -- Unprecedented rapid...

Aravindhan Veerapandiyan, MD, Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital and EXPLORE44® trial investigator, will present topline del-zota data from Phase 1/2 EXPLORE44 trial at 2025 MDA…