Oligonucleotide Chemistry

As an open and integrated platform, the nucleic acid chemical synthesis platform of Hitgen has leading technological, human-resource and platform-based advantages. It can meet the diverse needs of different market environments and various partners, empowering the R&D of nucleic acid drugs!

• A professional team for nucleic acid monomer modification and nucleic acid synthesis.

• Equipped with leading large-scale and high-throughput nucleic acid synthesizers and supporting equipment.

• Capable of producing nucleic acids ranging from micrograms to hundreds of grams, meeting the drug R&D requirements at different stages.

• With a complete range of preparative chromatography columns, it can quickly and efficiently develop purification methods.

• A strict quality control system, including analysis methods such as high - throughput next - generation sequencing, UPLC-MS detection, and real-time fluorescence quantitative detection, can fully guarantee product quality.

A large inventory of monomers (including modified monomers) can quickly meet customers' needs.

Research Background

RNA interference (RNAi) is a process of sequence - specific post - transcriptional gene silencing triggered by small interfering RNAs (siRNAs). Over the past 20+ years, RNAi technology has rapidly become a powerful tool for gene function research (such as loss-of-function and target validation tests), and a potential therapeutic agent for treating various human diseases, including genetic disorders, cardiovascular diseases, infectious diseases, cancers, immune-related diseases, etc. Therapeutic agents based on the RNAi principle of genes offer high specificity and can precisely target human diseases (even "undruggable" targets) and viral genomes. To date, four siRNA drugs have been approved globally for genetic diseases and hypercholesterolemia.

Business Scope of Hitgen Nucleic Acid Drugs

The expert team for nucleic acid drug R&D at Hitgen covers several key areas: bioinformatics, computer science, organic chemistry, RNA biology, molecular and cell biology, nucleic acid drug chemistry, translational research, and clinical medicine, etc. We can provide highly customized RNAi services, offering all - round support for our clients' RNA drug R&D journey. For example, efficient siRNA design, off - target effect prediction algorithms, design, synthesis and biological function evaluation of chemically modified siRNAs. The obtained siRNA molecules demonstrate reliable high - potential and specific gene - silencing effects, with higher stability and less cytotoxicity. We have also established various supporting functional platforms to enhance the project process and accelerate the timeline at each stage of RNA drug development.

R&D Directions and Plans for Hitgen's Nucleic Acid Drugs

Currently, the nucleic acid drug R&D at Hitgen focuses on human malignant solid tumors and immune-related diseases. We conduct target analysis, design, synthesis and screening of efficient siRNA molecules for tumor-and immune-regulation-related targets, and use various in-vitro models (cell lines and primary cells) and in - vivo models (mice and rats) to verify and evaluate the mechanism of action, therapeutic activity, biodistribution and tissue toxicity of nucleic acid drugs, etc. The research content and technologies include (but are not limited to):

• RNA interference technology (RNAi): Gene therapy mediated by small interfering RNAs / microRNAs (siRNA / microRNA).

• RNA activation technology (RNAa): Gene regulation and therapy mediated by small activating RNAs (saRNA).

• Systematic Evolution of Ligands by Exponential Enrichment (SELEX): Aptamer - mediated targeted disease treatment, as well as drug tracking and delivery systems.

• RNA delivery systems: Receptor - specific and liposome nanoparticle - mediated gene delivery.

• Molecular biology techniques: qRT-PCR, Taqman PCR, 5'-RACE PCR, next - generation sequencing (RNA - seq), SPR, ITC, in-vitro transfection, electrophoresis, flow cytometry, confocal microscopy, Western/Northern Blotting, ELISA, etc.

• Cell culture techniques: Isolation, culture and differentiation of cell lines and primary cells, etc.

Animal models: Establishment and characterization of mouse models with tumor cell scaffolds (CDX orthotopic and subcutaneous), construction of clinically relevant disease models (PDX), evaluation and verification of the therapeutic activity, biodistribution and tissue toxicity of small molecules and nucleic acid drugs, etc.