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drisapersen
- Development stage
- Discontinued
- Lead developer
- BioMarin
- 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
Drisapersen is an investigational antisense oligonucleotide designed for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. It is a 2'-O-methyl phosphorothioate oligonucleotide that binds to dystrophin pre-mRNA and induces skipping of exon 51 during splicing. This restores the reading frame in certain DMD mutations and enables production of a partially functional dystrophin protein. Drisapersen was originally developed by Prosensa and later acquired by BioMarin for further development. The drug targets approximately 13% of DMD patients with specific deletions suitable for exon 51 skipping[3][4][5][6].
- Brand names
- Kyndrisa
- Other names
- drisapersenPRO051PRO-051PRO 051GSK2402968GSK-2402968GSK 2402968Kyndrisa
02
Targets
DMD (Dystrophin)
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