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Drug intelligence / Profile preview
delpacibart braxlosiran (Novartis)
- Development stage
- Unknown
- Lead developer
- Novartis
- 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, Monoclonal Antibodies → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
Delpacibart braxlosiran (del-brax, formerly AOC 1020) is an investigational antibody-oligonucleotide conjugate (AOC) designed to treat the underlying cause of facioscapulohumeral muscular dystrophy (FSHD). It consists of a humanized monoclonal antibody targeting transferrin receptor 1 (TfR1) conjugated via an MCC maleimide linker to a small interfering RNA (siRNA) payload, siDUX4.6, which targets double homeobox 4 (DUX4) mRNA. By binding to TfR1, the conjugate is internalized into skeletal muscle cells, where the siRNA payload mediates the degradation of DUX4 mRNA, thereby suppressing the toxic expression of DUX4 protein. Originally developed by Avidity Biosciences, the program was acquired by Novartis in February 2026 and is currently in Phase 3 clinical development.
- Other names
- DUX4 AOCDUX-4 AOCDUX 4 AOCDUX4 AOCsDUX-4 AOCsDUX 4 AOCsdel-brax
02
Targets
TFRC (Transferrin Receptor)
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