Nucleic Acid Drug Developer

Therapeutic Nucleic AcidsWith tremendous potential to revolutionize vaccination, gene therapy, cell therapy, and the treatment of cancer and hereditary diseases. Since the first clinical studies on therapeutic nucleic acids began in the 2000s, significant progress has been made in the design, modification, and delivery of nucleic acid molecules. Various nucleic acid-based therapies have been studied, including mRNA, siRNA, plasmid DNA, viral and bacterial vectors, and patient-derived cell therapies, more than a dozen of which have been approved by the U.S. Food and Drug Administration.(FDA)Approved for human use and more are in clinical trials. However, nucleic acid therapeutics still face multiple challenges, such as off-target effects, unwanted immune stimulation, and low delivery efficiency. For mRNA-based therapies, the rapid degradation of mRNA by nucleases in the serum makes the delivery process even more challenging.
The current LNP delivery system typically includes cationic lipids, phospholipids, cholesterol, and PEG-lipid conjugates.Four-Lipid Component LNP Formulation(For example, a typical lipid ratio with cationic lipid: cholesterol: DSPC: DSPC: DMG-PEG = 50:38.5:10:1.5)It has been proven successful in a variety of commercial drug products, but there is still room for improvement. There remains a need to develop simplified lipid nanoparticle compositions without sacrificing the delivery efficiency of nucleic acid payloads.
On March 18, 2025, the China National Intellectual Property Administration disclosedAbogen BiosciencesApplied for“Composition of Lipid NanoparticlesPatent(Publication No.: CN119654141A)。

This patent provides aThreeLNP Formulation of Lipid Components((Without phospholipid components, or with reduced/insignificant amounts of phospholipid components). The three-lipid LNP formulation was administered via tail vein injection andIntramuscular injection experiments can enhance gene expression levels in mouse models.About2x。
At the same expression level, compared to conventional four-lipid LNP formulations, AbogenThree-Lipid LNP Formulation In VivoInduces relatively less IL-6 production, with milder side effects.For intravenous injection, the three-lipid LNP formulation group showedLower maximum weight loss and faster weight regain(100% body weight restored within two days). Using a tri-lipid LNP formulation(L-50/1.5 and L-60/1.5)The treatment group of mice after intramuscular administrationLess time is needed to restore its body weight., proving that the formulationHas a certain level of biosafety.。
About Abogen
Abogen Biosciences (referred to as "Abogen") is an innovative biopharmaceutical company specializing in the research and development of messenger ribonucleic acid (mRNA) drugs. The company owns industry-leading, proprietary intellectual property rights over its mRNA and nanodelivery technology platforms. It is one of the few mRNA drug research and development enterprises in China with complete value chain capabilities, covering all aspects from mRNA design, formulation development, to large-scale production. The company has established a rich product pipeline, spanning multiple fields including infectious disease prevention and treatment, cancer immunotherapy, protein replacement therapy, and mRNA COVID-19 vaccines.

