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A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

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Trial Profile

A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 20 Feb 2026

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At a glance

  • Drugs Delpacibart etedesiran (Primary)
  • Indications Myotonic dystrophy
  • Focus Adverse reactions; First in man; Proof of concept
  • Acronyms MARINA
  • Sponsors Avidity Biosciences

Most Recent Events

  • 18 Feb 2026 Results presented in the Avidity Biosciences Media Release.
  • 18 Feb 2026 According to an Avidity Biosciences media release, final results from this completed study will be published in the February 19 issue of The New England Journal of Medicine (NEJM). Nicholas E. Johnson, M.D., M.Sci., FAAN, professor and vice chair of research in the Department of Neurology at Virginia Commonwealth University, and primary investigator of the MARINA trial- is the lead author.
  • 27 Feb 2025 According to an Avidity Biosciences media release, publication of data analyses from the completed Phase 1/2 MARINA trial in 2025.

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