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OliPass Dystrophin PNA
- Development stage
- Preclinical
- Lead developer
- OliPass
- Modality
- 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
- Administration
- Subcutaneous, Intravenous, Intraperitoneal, Intramuscular, Oral, Ophthalmic, Intradermal, Intraocular, Intracerebral, Inhalation, Intranasal, Rectal, Topical
01
Overview
OliPass Dystrophin PNA is an investigational antisense oligonucleotide (ASO) therapy being developed by OliPass for the treatment of Duchenne Muscular Dystrophy (DMD). The drug utilizes OliPass's proprietary Peptide Nucleic Acid (PNA) platform, known as OPNA, which is engineered to enhance cell permeability and binding affinity compared to traditional ASOs. Its primary mechanism of action is to induce exon skipping during the splicing of dystrophin pre-mRNA. By bypassing specific mutated exons, the therapy aims to restore the reading frame of the dystrophin gene, enabling the production of a truncated but functional dystrophin protein. This approach is intended to slow the progressive muscle degeneration characteristic of DMD. The program is currently in the research and preclinical development stage.
- Other names
- OliPass DMD programDystrophin PNAOliPass-Dystrophin-antisense oligonucleotide (PNA)-Duchenne Muscular Dystrophy (DMD)
02
Targets
Neuraminidase
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