ArticleNucleic acids research2026
Development of a DUX4-targeting antibody oligonucleotide conjugate as a therapy for FSHD.
Article in Nucleic acids research, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
2 citing papers in PubMed.
- RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies.Biomolecules · 2026Review
- Evolution of siRNA Therapeutics: From Mechanistic Foundations to Clinical Expansion.Pharmaceutics · 2026Review
Corrections and comments
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Authors and funding
16 authors.
Funding
Abstract
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscular disease in which genetic mutations activate DUX4 expression in skeletal muscle. Currently, there are no approved therapies for FSHD. We developed Delpacibart braxlosiran (del-brax, also known as AOC 1020), an antibody oligonucleotide conjugate (AOC), for the treatment of FSHD that is designed to specifically target and reduce DUX4 mRNA in skeletal muscle. AOC 1020 is composed of DUX4 mRNA-targeting small interfering RNA (siRNA), siDUX4.6, conjugated to a human transferrin receptor 1 (TfR1)-targeting monoclonal antibody to facilitate productive siRNA delivery to muscle. We demonstrate that siDUX4.6 reduces DUX4-regulated gene expression in FSHD patient-derived myotubes in vitro and in skeletal muscle of the ACTA1-MCM; FLExDUX4 FSHD mouse model in vivo. Single systemic intravenous treatment was sufficient to prevent DUX4-induced muscle weakness and fibrosis in this FSHD mouse model and reduce DUX4-regulated genes by ∼75% 8 weeks post-dose. The pharmacokinetic profiles of AOCs with siDUX4.6 were comparable in murine and non-human primate muscle. These data demonstrate the potential of AOC 1020 to treat the underlying cause of FSHD by suppressing DUX4 expression in muscles of patients with FSHD. The safety and efficacy of AOC 1020 is currently being investigated in clinical trials.
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Registered trials
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