2409088-11-3
Chemical Structure
Brogidirsen
Synonym(s): NS 089; NCNP 02
- CAS No.: 2409088-11-3
- Formula:C280H440N119O105P23
- Molecular Weight:7865.60
IUPAC Name: N-(9-((2R,3R,4R,5R)-3-((tert-butyldimethylsilyl)oxy)-4-hydroxy-5-(hydroxymethyl)tetrahydrofuran-2-yl)-9H-purin-6-yl)benzamide
SMILES: [Brogidirsen]
Biological Activity: Brogidirsen (NS 089; NCNP 02) is a dual-targeting antisense oligonucleotide capable of inducing AAexon skipping. Brogidirsen consists of two 12-mer phosphorodiamidate morpholino oligomers (PMOs) linked directly together. Through these PMOs, Brogidirsen targets regenerating muscle fibers, facilitating the reprogramming of cells that overexpress MYOD1 into the myogenic lineage. Brogidirsen is used in research related to Duchenne muscular dystrophy[1][2][3].
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Brogidirsen | Brogidirsen (NS 089; NCNP 02) is a dual-targeting antisense oligonucleotide capable of inducing AAexon skipping. Brogidirsen consists of two 12-mer phosphorodiamidate morpholino oligomers (PMOs) linked directly together. Through these PMOs, Brogidirsen targets regenerating muscle fibers, facilitating the reprogramming of cells that overexpress MYOD1 into the myogenic lineage. Brogidirsen is used in research related to Duchenne muscular dystrophy. | |||||||||||||||||||||
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References
- [1]. Aoki Y, et al. Brogidirsen-The First Dual-Targeting Exon 44 Skipping Drug for Duchenne Muscular Dystrophy: Safety, Tolerability, and High Dystrophin Restoration in Long-Term Clinical Studies[C]//MOLECULAR THERAPY. 50 HAMPSHIRE ST, FLOOR 5, CAMBRIDGE, MA 02139 USA: CELL PRESS, 2025, 33(4).
- [2]. Komaki H, et al. Phase 1/2 trial of brogidirsen: Dual-targeting antisense oligonucleotides for exon 44 skipping in Duchenne muscular dystrophy. Cell Rep Med. 2025;6(1):101901. [Content Brief]
- [3]. Chassin A, et al. RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models[J]. International Journal of Molecular Sciences, 2026, 27(6): 2755.
