Accelerated approval path confirmed for del-zota; Biologics License Application (BLA) submission planned for year end 2025 On track to complete enrollment in...

Today, Avidity Biosciences announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of delpacibart zotadirsen (del-zota) for individuals living with Duchenne who are amenable...

ENTR-601-44 didn’t elevate dystrophin levels to analyst expectations, or to the baseline change recorded by Novartis’ del-zota.

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...

Avidity Biosciences, Inc. has announced its Managed Access Program (MAP) for delpacibart zotadirsen (del-zota) for eligible individuals living with Duchenne who are amenable to exon 44 skipping in the United States. MAPs allow a limited...

Yesterday, Avidity Biosciences announced a definitive agreement to be acquired by Novartis. The transaction includes Avidity’s late-stage programs, including delpacibart zotadirsen (del-zota) for Duchenne. According to Avidity’s announcement, the company and Novartis intend to keep...

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...

In the trials, Avidity's DMD drug del-zota demonstrated up to 25% increase of dystrophin.

Reported Monday, Oct 13 - 9:02am EDT (TheFly subscription required to read more.)

Reported Wednesday, Sep 10 - 9:46am EDT (TheFly subscription required to read more.)

Reported Monday, Mar 17 - 7:09am 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 has announced a Managed Access Program for del-zota, an innovative therapy for Duchenne Muscular Dystrophy, aimed at supporting patients with specific gene mutations.

Avidity Biosciences announces successful pre-BLA meeting with FDA, paving the way for del-zota submission addressing Duchenne muscular dystrophy in 2026.

Avidity Biosciences showcases data on Del-Zota's effects on DMD44 at the 30th World Muscle Society Congress, highlighting breakthroughs.

Avidity Biosciences' recent trials on Del-zota exhibit significant improvements in patients with Duchenne muscular dystrophy, paving the way for future treatment options.