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AOC 1024
- Development stage
- Unknown
- Lead developer
- Avidity Biosciences
- Modality
- 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, Monoclonal Antibodies → Antibody-Based Therapeutics, Antibody Conjugates → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
AOC 1024 is an investigational antibody oligonucleotide conjugate (AOC) designed for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Developed by Avidity Biosciences (and part of a planned acquisition by Novartis), AOC 1024 utilizes a proprietary platform to deliver a therapeutic payload directly to skeletal muscle. The drug consists of a monoclonal antibody that targets the transferrin receptor 1 (TfR1), which is highly expressed on muscle cells, conjugated to a small interfering RNA (siRNA). This siRNA is specifically engineered to target and degrade the mRNA of the Double Homeobox 4 (DUX4) gene. In FSHD, the aberrant expression of DUX4 in muscle tissue leads to progressive muscle cell death and weakness; by silencing DUX4, AOC 1024 aims to halt disease progression and preserve muscle function. It is currently being evaluated in clinical trials to assess its safety, tolerability, and impact on DUX4 expression and muscle biomarkers.
- Other names
- delpacibart braxosiran
02
Targets
TFRC (Transferrin Receptor)DUX4 (Double Homeobox 4)
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