On April 22, 2024 Vect-Horus, a privately held biotechnology company that designs and develops molecular vectors that facilitate the targeted delivery of therapeutic molecules and imaging agents, reported the publication of a paper in the open-access scientific journal Pharmaceutics Vol 26 (MDPI) (Press release, Vect-Horus, APR 22, 2024, View Source [SID1234642207]).
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The paper, entitled "LDLR-Mediated Targeting and Productive Uptake of siRNA-Peptide Ligand Conjugates In Vitro and In Vivo", outlines a study which showed how peptides developed by the company, that target the low-density lipoprotein receptor (LDLR) can support delivery of a model short interfering RNA (siRNA), both in vitro and in vivo. Because the LDLR is differentially expressed in organs and is overexpressed in many cancers including glioblastoma multiforme (GBM) and pancreatic ductal adenocarcinoma (PDAC), the results open new opportunities for delivery to different organs or tumors.
A vector belonging to this family of LDLR-targeting peptides is applied as a theragnostic agent for the diagnostic of GBM and PDAC. The agent, currently in early Phase 1 clinical trials, is co-developed with RadioMedix.
"These results reinforce the potential of vectors to significantly improve the transport and delivery of therapeutic and imaging agents to improve the diagnostic and treatment of cancer and CNS disorders," said Alexandre Tokay co-founder and CEO of Vect-Horus. "This provides further impetus to our current partnerships and builds on other recent licensing agreements, with Novo Nordisk and Ionis Pharmaceuticals."
siRNAs are promising therapeutic agents because of their specificity and their potential to modulate gene expression, but their delivery into cells and tissues of interest remains highly challenging due to the lack of efficient and selective delivery systems.
The study identified and optimized a family of peptide-based vectors that target the LDLR. The results validated the LDLR-binding peptides as viable ligands which can trigger efficient delivery of therapeutic oligonucleotides, both in a cellular model and in vivo after systemic administration.