Avidity Biosciences: FDA Grants Orphan Drug Designation To AOC 1044

(RTTNews) - Avidity Biosciences, Inc. (RNA) announced the FDA has granted Orphan Drug designation to AOC 1044, the company's investigational therapy in development for the treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping. AOC 1044 is being assessed in the phase 1/2 EXPLORE44 clinical trial for people living with DMD44.

Avidity plans to share results from the healthy volunteer portion of the EXPLORE44 trial in the fourth quarter of 2023.

In April 2023, AOC 1044 received FDA Fast Track designation for the treatment of DMD44.

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