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delpacibart etedesiran
- Development stage
- Phase 3
- Lead developer
- Avidity Biosciences
- 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 etedesiran** is an investigational antibody oligonucleotide conjugate developed by Avidity Biosciences and now controlled by Novartis for the treatment of myotonic dystrophy type 1. Also known as del-desiran and formerly AOC 1001, it combines a humanized monoclonal antibody targeting transferrin receptor 1 with a small interfering RNA directed against toxic DMPK messenger RNA. Transferrin receptor 1-mediated uptake is intended to deliver the siRNA into skeletal, cardiac, and smooth muscle, where RNA interference reduces DMPK mRNA and may alleviate downstream RNA missplicing associated with disease pathology. Delpacibart etedesiran is administered by intravenous infusion and remains in Phase 3 development in the HARBOR trial. ([aviditybiosciences.com](https://www.aviditybiosciences.com/pipeline/dm1))
- Other names
- del-desiran
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)TFRC (Transferrin Receptor)Myotonic dystrophy protein kinase messenger RNA
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