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PMO44
- Development stage
- Unknown
- Lead developer
- Avidity Biosciences
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
PMO44 is a phosphorodiamidate morpholino oligomer (PMO) designed to treat Duchenne muscular dystrophy (DMD) in patients with deletions amenable to exon 44 skipping. As an antisense oligonucleotide, PMO44 binds to the dystrophin pre-mRNA and induces the skipping of exon 44 during the splicing process. This mechanism restores the mRNA reading frame, enabling the production of a truncated but functional dystrophin protein in skeletal and cardiac muscle tissues. PMO44 is the therapeutic oligonucleotide payload of AOC 1044 (del-zota), an antibody-oligonucleotide conjugate developed by Avidity Biosciences. In this conjugate, PMO44 is linked to a monoclonal antibody targeting the transferrin receptor 1 (TfR1) to enhance its delivery and uptake into muscle cells.
- Other names
- phosphorodiamidate morpholino oligomer 44
02
Targets
DMD (Dystrophin)
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