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RX-0201-nano
- Development stage
- Unknown
- 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, Liposomes → Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
**RX-0201-nano** is a nanoliposomal formulation of the antisense oligonucleotide RX-0201, designed to inhibit Akt1 (protein kinase B) expression by targeting its mRNA, thereby reducing cancer cell growth and proliferation. Developed initially by Rexahn Pharmaceuticals with nanoparticle delivery technologies licensed from Ohio State University (LCAN) and in collaboration with Zhejiang Haichang Biotechnology (QTsome) for hepatocellular carcinoma, it aims to improve nuclear delivery, tumor targeting (e.g., via folate receptor), and efficacy over the parent RX-0201 (Archexin®). It has been advanced to preclinical stages and planned for Phase IIa proof-of-concept in liver cancer, with prior RX-0201 data showing safety in Phase I/II trials for solid tumors.[1][2][4][5][6][7]
- Brand names
- Archexin
- Other names
- RX-0201RX0201RX 0201
02
Targets
AKT1 (Proto-oncogene serine/threonine-protein kinase Akt1)
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