Nucleic Acid and Gene Therapy Bioanalysis

Specialised bioanalytical and characterization services for nucleic acid therapeutics and gene therapy programmes.

Nucleic acid therapeutics and gene therapy vectors represent some of the most analytically demanding drug modalities in modern pharmaceutical development. From antisense oligonucleotides (ASOs) and RNA interference (RNAi) therapeutics to mRNA therapeutics, viral vectors, and plasmid DNA, these advanced modalities require highly specialised bioanalytical platforms and extensive expertise in the applicable regulatory frameworks.

AMSbiopharma provides a comprehensive portfolio of bioanalytical and characterization services for nucleic acid therapeutics, supporting programmes from early preclinical pharmacokinetic/toxicokinetic (PK/TK) studies through CMC characterization and regulatory submissions.

All bioanalytical studies are performed under controlled quality systems, with method development and validation conducted in accordance with ICH M10 guidelines.

Our catalog of services include

Metabolomics applications

PK/PD Analysis of Oligonucleotides, Transgenes and Viral DNA/RNA / NON GLP

The bioanalysis of therapeutic oligonucleotides and gene therapy vectors in biological matrices requires analytical approaches with significantly greater complexity and sensitivity than those used for conventional small-molecule drugs. The structural similarity between the active therapeutic and endogenous nucleic acid species places exceptional demands on method sensitivity, specificity, and selectivity.

AMSbiopharma provides validated PK/PD bioanalytical services for oligonucleotide therapeutics, supporting preclinical and translational development programmes with robust quantitative data generated using state-of-the-art bioanalytical methodologies.

Key analytical capabilities

  • Validated bioanalysis of antisense oligonucleotides (ASOs), RNA interference (RNAi) therapeutics, mRNA therapeutics, and viral DNA/RNA in biological matrices 
  • Bioanalytical capabilities for plasma, serum, tissue, and cerebrospinal fluid (CSF) 
  • ICH M10-compliant method development and validation 
  • Analytical support for tissue distribution and biodistribution studies

Reliable quantification of nucleic acid species and the assessment of gene expression changes in biological matrices are critical analytical requirements for the development of gene therapies and RNA-based therapeutics.

AMSbiopharma provides validated bioanalytical services for the quantification of DNA and RNA targets in biological matrices, including the assessment of gene expression changes relevant to pharmacodynamic monitoring and safety evaluation. Method development and validation are performed in accordance with applicable regulatory guidelines, ensuring robust, reproducible, and regulatory-ready data.

Key analytical capabilities

  • Quantification of DNA and RNA targets in plasma, tissue, and other biological matrices 
  • Gene expression analysis to support pharmacodynamic biomarker programmes 
  • Method development and validation in accordance with applicable regulatory guidelines 
  • Analytical support for preclinical development programmes 
  • Applicable to RNA therapeutics and gene therapy vector programmes 
  • Comprehensive documentation to support regulatory submissions

Genetic biomarkers play an increasingly important role in modern drug development, providing mechanistic and predictive insights that support patient stratification, pharmacodynamic monitoring, and precision medicine approaches.

AMSbiopharma provides validated analytical services for the detection and quantification of genetic biomarkers in biological samples. Our methods are developed for regulatory use in both preclinical and clinical studies, with biomarker strategies aligned with current regulatory qualification guidelines.

Key analytical capabilities

  • Detection and quantification of genetic biomarkers in biological samples 
  • Biomarker strategies aligned with regulatory expectations and qualification frameworks 
  • Applicable to oncology, rare diseases, and gene therapy programmes 
  • Method development for preclinical studies

Plasmid DNA is a critical component of the drug substance used in the manufacture of gene therapies and DNA vaccines. Plasmid quality, integrity, and isoform distribution are critical quality attributes (CQAs) that must be characterised and controlled throughout the manufacturing process. Regulatory authorities require comprehensive plasmid characterization data as part of the CMC package for gene therapy products.

AMSbiopharma provides validated analytical services for plasmid DNA isoform analysis, enabling the accurate quantification and relative distribution of supercoiled, open circular, and linear plasmid isoforms in both drug substance and drug product samples.

Key analytical capabilities

  • Quantification and relative distribution of plasmid DNA isoforms (supercoiled, open circular, and linear) 
  • Analytical support for in-process quality control and drug substance testing 
  • Method development to support CMC development and process optimisation 
  • Applicable to gene therapy vectors and DNA vaccine development programmes 
  • Method development with appropriate sensitivity and specificity