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renadirsen
- Development stage
- Phase 2
- Lead developer
- Daiichi Sankyo
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Renadirsen (DS-5141b) is an antisense oligonucleotide drug developed for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 45 skipping. It utilizes a proprietary ethylene-bridged nucleic acid (ENA) oligonucleotide technology, which binds to exon 45 of the dystrophin pre-mRNA, inducing its skipping during mRNA splicing. This process restores the reading frame and enables production of a truncated but functional dystrophin protein, similar to that seen in Becker muscular dystrophy. Renadirsen was designed for subcutaneous administration and aimed to improve both skeletal and cardiac muscle function by increasing exon skipping efficiency compared to earlier chemistries such as PMOs. The drug was co-developed by Daiichi Sankyo and the Orphan Disease Treatment Institute[3][5][6][7][8].
- Other names
- renadirsen
02
Targets
DMD (Dystrophin)
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