Gene Therapy Preclinical Studies

Regulatory guidelines for gene therapy toxicology and safety pharmacology studies that must be conducted to advance new therapies to human clinical trials are still evolving, and unlike other therapy classes such as small molecules or biologics, gene therapy preclinical study requirements are individually tailored to the characteristics of the therapy. In addition, gene therapy includes diverse approaches including ex vivo gene editing, and in vivo viral and non-viral vector treatments.

IITRI offers an experienced toxicology team to provide expert assistance in planning and executing gene therapy preclinical studies and programs for IND submissions. We have been designing and executing GLP-compliant preclinical studies in compliance with FDA requirements since these guidelines were first instituted in 1979. Our supporting technologies in bioanalytical methods and expertise in viral vector biology and molecular biology forms the foundation of our gene therapy study team.

Gene Therapy Vector Types

  • Viral (lentivirus, adenovirus, AAV)
  • Naked DNA, nanoparticles, RNA

Gene Therapy Preclinical Study Types

  • Proof of concept (efficacy or gene expression studies)
  • Biodistribution (vector and/or gene product quantitation in blood and/or tissues)
  • GLP toxicology and safety

Toxicology Study Bioanalysis Assays

  • Real-time qPCR (DNA), real-time RT-qPCR (RNA)
  • ELISA - anti-drug antibody analysis
  • ELISpot - cytokine-secreting cell quantitation assay
  • Luminex - biomarker profiling

All animal species (rodent, nonrodent, NHP)
All routes of administration (including inhalation and electroporation)