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LHHK LNP
- Development stage
- Preclinical
- Lead developer
- University of Nebraska Medical Center
- 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
01
Overview
LHHK LNP is an experimental lipid nanoparticle (LNP) delivery system designed for the systemic delivery of small interfering RNA (siRNA) to treat various cancers. The platform is based on a novel biodegradable lipid, LHHK, which features a headgroup modified with the amino acids histidine and lysine. This specific modification provides an optimal pKa of 6.08, which enhances endosomal escape and cellular uptake while minimizing systemic toxicity. In preclinical studies, LHHK LNPs have been successfully used to deliver siRNA targeting IKKα in prostate cancer and IKBKE in pancreatic cancer, resulting in significant gene silencing, inhibition of cell proliferation, and suppression of tumor growth in mouse models.
- Other names
- LHHK lipid nanoparticle
02
Targets
IKBKE (IκB kinase epsilon)Inhibitor of nuclear factor kappa-B kinase subunit alpha mRNA
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