Drug intelligence / Profile preview

LCAN-RX-0201

Development stage
Preclinical
Lead developer
Opus Genetics
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

LCAN-RX-0201 is a nanoparticle-based delivery system for RX-0201 (archexin), a 20-mer phosphorothioate antisense oligonucleotide that targets Akt-1 (protein kinase B alpha). The formulation consists of lipid-coated albumin nanoparticles (LCAN) composed of DOTAP, soyPC, TPGS, and a human serum albumin-pentaethylenehexamine (HSA-PEHA) conjugate. A folate-targeted version, F-LCAN-RX-0201, incorporates folate-PEG-DSPE to specifically target folate receptor-expressing tumor cells. By encapsulating the antisense agent, the LCAN system improves the pharmacokinetic profile, increases cellular uptake, and enhances the antitumor activity of RX-0201 compared to the free oligonucleotide. It was developed by Rexahn Pharmaceuticals in collaboration with researchers at The Ohio State University.

Other names
folate receptor-targeted lipid coated albumin nanoparticles containing RX-0201
02

Targets

AKT1 (Proto-oncogene serine/threonine-protein kinase Akt1)

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