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PCSK9 antisense oligonucleotide
- 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
- Oral
01
Overview
PCSK9 antisense oligonucleotide is a preclinical-stage RNA therapeutic being developed by OliPass Corporation for the treatment of hypercholesterolemia. The drug utilizes OliPass's proprietary OliPass Peptide Nucleic Acid (OPNA) technology, which features chemically modified PNAs designed to significantly enhance cell permeability and RNA binding affinity. Unlike many antisense oligonucleotides that require subcutaneous or intravenous injection, this candidate is specifically engineered for oral administration, leveraging the high potency and stability of the OPNA platform to achieve therapeutic levels in the liver. The drug functions through an exon-skipping mechanism, binding to PCSK9 pre-mRNA to modulate gene expression and reduce the production of the PCSK9 protein. By inhibiting PCSK9, the therapeutic increases the availability of LDL receptors on the surface of liver cells, leading to improved clearance of LDL cholesterol from the blood.
- Other names
- OliPass PCSK9 inhibitorOPNA PCSK9 antisense oligonucleotide
02
Targets
PCSK9 (Proprotein convertase subtilisin/kexin type 9)
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