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HARBOR-OLE Long-Term Safety Study of AOC 1001 in Myotonic Dystrophy Type 1
jRCT2021250036Phase 3Active, not recruiting
- Sponsor
- Avidity Biosciences
- Start
- Dec 2025
- Modality
- Antibody Conjugates → Antibody-Based Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
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Overview
Phase 3, open-label, single-arm trial of delpacibart etedesiran in myotonic dystrophy type 1 (DM1), sponsored by Avidity Biosciences. Primary endpoint: Treatment-emergent adverse events. Started Dec 2025; status: active, not recruiting.
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Drugs
delpacibart etedesiran
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Conditions
myotonic dystrophy type 1 (DM1)
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