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Trial intelligence / Profile preview
SAFARI44: AOC 1044 in Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping
2025-523087-20-00Phase 3Authorised
- Sponsor
- Avidity Biosciences
- Start
- Jul 2026
- Modality
- Antibody Conjugates → Antibody-Based Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 10
01
Overview
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing AOC 1044 arm with Placebo arm (2 arms). Primary endpoint: Change from baseline to Week 54 in TTR velocity. Planned start Jul 2026; status: authorised.
02
Drugs
delpacibart zotadirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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