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BMN-053
- Development stage
- Discontinued
- Lead developer
- BioMarin
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
BMN-053 is an experimental antisense oligonucleotide drug developed for the treatment of Duchenne muscular dystrophy (DMD), specifically targeting patients with mutations amenable to exon 53 skipping. The drug operates via an exon-skipping mechanism using a phosphorodiamidate morpholino oligomer (PMO) backbone to bind pre-mRNA and induce skipping of exon 53 during mRNA splicing. This restores the reading frame of the dystrophin gene transcript, enabling production of a truncated but functional dystrophin protein similar to that seen in Becker muscular dystrophy. The primary developer was BioMarin Pharmaceutical. Clinical development included Phase I/II trials but has since been discontinued[1][2][3][4][5][7].
- Other names
- PRO-053PRO053PRO 053
02
Targets
Neuraminidase
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