HitGen offers comprehensive solutions for the development of oligonucleotide therapeutics, covering the entire process from research and development to production.
Precise and Efficient In Vitro Activity Evaluation System
• Multi-Model Validation
Compatible with immortalized cell lines, primary cells, and other models, supporting diverse nucleic acid therapeutics including mRNA, siRNA, ASO, Dual-targeting siRNA, and more.
• Multi-Dimensional Analysis
Gene Expression Modulation:
Quantify mRNA knockdown efficiency of target genes via qPCR and RNA-Seq.
Protein Functional Validation:
Accurately detect changes in target protein expression using ELISA, Western Blot, and flow cytometry.
Systematic Druggability Assessment Matrix
Evaluation Dimension | Technical Methods | Significance |
Stability | Serum/plasma stability assays, accelerated degradation testing | Optimizing chemical modification strategies to extend in vivo half-life. |
Pharmacokinetics | LC-MS/MS quantification, tissue distribution analysis | Deciphering absorption, distribution, and metabolism patterns to guide formulation design. |
Immunogenicity | TLR pathway activation assays, cytokine release profiling | Mitigating immunotoxicity risks of nucleic acid drugs. |
Cytotoxicity | CCK-8 assays, LDH release tests, mitochondrial membrane potential detection, Caspase-dependent apoptosis analysis | Evaluating safety profiles and screen candidates with high therapeutic indices. |
Off-Target Effects | Whole-transcriptome sequencing (RNA-Seq), bioinformatics prediction | Filtering off-target risks and enhance drug specificity. |
Differentiated Technical Advantages
• High-Sensitivity Detection:
Capable of detecting nucleic acid drug activity as low as 0.1 pM, enabling development for low-abundance targets.
• Cross-Species Compatibility:
Covers human, murine, and non-human primate models for seamless preclinical-to-clinical translation.
• Customized Services:
Tailor evaluation protocols based on drug mechanisms of action (MOA) and project requirements.
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