Avidity Biosciences (RNA) Announces Positive One-Year Data For its, Del-Zota

Avidity Biosciences, Inc. (NASDAQ:RNA) is one of the Best Performing Long Term Stocks According to Analysts. On September 10, Avidity Biosciences, Inc. (NASDAQ:RNA) announced positive one-year data for its, del-zota, in the EXPLORE44 and EXPLORE44-OLE trials for Duchenne muscular dystrophy patients amenable to exon 44 skipping.

Management noted that the data show a reversal of disease progression and significant functional improvements compared to baseline and natural history. Del-zota works by delivering phosphorodiamidate morpholino oligomers to muscle and heart tissue to skip exon 44 in the dystrophin gene. This enables production of near-full-length dystrophin protein, crucial for muscle protection.

The result shows that the trial participants showed about a 25% increase in dystrophin and up to 58% restored total dystrophin levels. Moreover, Creatine kinase (CK), a marker of muscle damage, dropped by over 80% and was sustained near normal. Avidity Biosciences, Inc. (NASDAQ:RNA) plans to file a Biologics License Application (BLA) with the FDA by the end of 2025 for accelerated approval, marking a major step toward a new treatment option for this severe genetic disease.

Avidity Biosciences, Inc. (NASDAQ:RNA) is a biotech company focused on creating a new class of RNA therapies called Antibody Oligonucleotide Conjugates.

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Disclosure: None. This article is originally published at Insider Monkey.