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usiRNA targeting apolipoprotein B
- Development stage
- Discontinued
- Lead developer
- Marina Biotech
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
UsiRNA targeting apolipoprotein B is an experimental RNA interference (RNAi) therapeutic developed by Marina Biotech (formerly MDRNA, Inc.) for the treatment of hypercholesterolemia. The drug utilizes Marina Biotech's proprietary Unlocked Nucleobase-modified siRNA (usiRNA) technology, which incorporates unlocked nucleic acid (UNA) residues into the siRNA duplex. This modification is designed to enhance the stability of the siRNA, reduce off-target effects by minimizing passenger strand activity, and potentially decrease the immune response compared to standard siRNA. By targeting the mRNA of apolipoprotein B (ApoB), a critical structural component of low-density lipoprotein (LDL) and very-low-density lipoprotein (VLDL) particles, the drug aims to inhibit the synthesis and secretion of these atherogenic lipoproteins from the liver, thereby lowering circulating cholesterol levels.
- Other names
- unlocked nucleobase-modified siRNA targeting apolipoprotein BApoB-targeting usiRNA
02
Targets
APOB (Apolipoprotein B)
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