Gene Therapy Preclinical Studies

The recent FDA approval of the breakthrough CAR-T ex-vivo gene therapy for childhood leukemia has given the rapidly expanding field of gene therapy another boost. Regulatory guidelines for preclinical studies to move new therapies to human clinical trials are still evolving, and unlike other therapy classes such as small molecules or biologics, gene therapy preclinical study sets are individually tailored to the characteristics of the therapy.

IITRI offers an experienced toxicology team who has been designing and executing preclinical studies with FDA requirements in mind since the process was instituted in 1979. Our supporting technologies in bioanalytical methods and expertise in viral vector biology and molecular biology qualifies us to provide expert assistance in planning and executing gene therapy preclinical studies and programs for IND submissions.

Vector Types

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

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

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)