Nucleic acid drugs represent a cutting-edge class of therapeutics that regulate gene expression with high precision. Their short development cycles, broad therapeutic potential, and low risk of drug resistance make them especially promising for treating rare and genetic diseases.
With deep technical expertise and extensive project experience, InnoStar has successfully supported nearly 100 small nucleic acid drug programs and over 30 mRNA drug development projects. We strictly adhere to international regulatory standards including NMPA, FDA, and OECD, and offer full support for global multi-center applications.

InnoStar Nucleic Acid Drug Solutions
Early Screening & Property Evaluation
-Plasma stability assessment
-Liver S9/liver cell metabolic stability testing
-Monkey liver puncture technology for PK/PD correlation
-Tissue distribution studies (including target organs)
-Metabolite identification and characterization
-Genus-specific pharmacokinetics
-In vitro and in vivo pharmacology studies
-Toxicity screening in early discovery

Non-Clinical Pharmacodynamic Studies
-Evaluation of drug effects on cellular activity, function, protein, and mRNA levels
-siRNA target engagement and off-target effect analysis
-Animal models with high target gene homology for pharmacological validation
 

Non-Clinical Pharmacokinetic Studies
-Full characterization of PK profiles of nucleic acid drugs
-Customized PK study designs for antisense, siRNA, mRNA, and other oligonucleotide platforms
 

Non-Clinical Safety Evaluation
-General toxicology
-Safety pharmacology
-Genetic toxicology
-Developmental and reproductive toxicity (DART)
-Phototoxicity
-Dependence potential
-Carcinogenicity studies
-Juvenile animal studies
-Inhalation toxicity studies

 Clinical Biological Analysis
Leveraging advanced analytical platforms such as LC-MS/MS, ELISA, ECL, qPCR, and dPCR, InnoStar offers end-to-end clinical bioanalytical services:
-PK, ADA, PD & biomarker analysis
-Oligonucleotide bioanalysis via high-sensitivity LC-MS/MS or Hybridization ELISA
-Cationic lipid analysis with ultra-sensitive LC-MS/MS for delivery components
-Immunogenicity analysis for carriers and expressed proteins (ELISA, ECL, cell-based assays)
-PD/Biomarker quantification: cytokines, protein expression, etc.
-RISC-siRNA complex detection: RNA immunoprecipitationstem-loop RT-qPCR
-Target gene mRNA quantification: RT-PCR, dPCR, RNA-seq

Project Experience
•Nearly 100 small nucleic acid programs completed
•30+ mRNA drug development projects
•Support for IND, NDA, early-stage screening, and exploratory non-GLP studies
Representative Achievements
•Development of the first siRNA drug approved for clinical hyperlipidemia treatment in Australia and China
•Successful submission of the world’s first IV-administered self-replicating RNA therapy for tumors
 

Partner with InnoStar to accelerate your nucleic acid drug development— from early discovery through IND and beyond—backed by global standards, deep expertise, and proven success.