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PRO-040201
- Development stage
- Discontinued
- Lead developer
- Arbutus Biopharma
- 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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
PRO-040201 is a small interfering RNA (siRNA) therapeutic developed to target and knock down the expression of Apolipoprotein B (ApoB), a key protein involved in the metabolism of low-density lipoprotein cholesterol (LDL-C). By inhibiting ApoB production through RNA interference, PRO-040201 aims to reduce LDL-C levels in patients with hypercholesterolemia. The drug utilizes Stable Nucleic Acid Lipid Particle (SNALP) technology for systemic delivery. Developed by Tekmira Pharmaceuticals Corporation (now Arbutus Biopharma), it reached Phase 1 clinical trials for hypercholesterolemia but was discontinued after immune stimulation was observed at higher doses[1][2][4][5].
- Other names
- TKM-ApoBApoB SNALP
02
Targets
APOB (Apolipoprotein B)
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