Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
delpacibart zotadirsen
- Development stage
- Phase 3
- Lead developer
- Avidity Biosciences
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antibody Conjugates → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
Delpacibart zotadirsen (formerly AOC 1044) is an antibody-oligonucleotide conjugate (AOC) developed by Avidity Biosciences for the treatment of Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 44 skipping. The drug consists of a monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated to a phosphorodiamidate morpholino oligomer (PMO) antisense oligonucleotide. By targeting TfR1, the AOC facilitates the delivery of the PMO into skeletal muscle and heart tissue. Once inside the cells, the PMO induces skipping of exon 44 in the dystrophin pre-mRNA, restoring the reading frame and allowing for the production of a truncated but functional dystrophin protein. It is currently in Phase 1/2 clinical development (EXPLORE44 trial).
- Brand names
- del-zota
- Other names
- delpacibart zotadirsen
02
Targets
TFRC (Transferrin Receptor)DMD (Dystrophin)
Beyond the preview
Go deeper on delpacibart zotadirsen.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on delpacibart zotadirsen.