The Preclinical Analytics Core provides skilled expertise in the evaluation of the biodistribution/shedding and of the expression profiles following in vivo administration of gene therapy products. Such analysis are required to understand the pharmacology and the pharmacokinetic of a therapeutic product, i.e. to determine the presence, the persistence and the expression level of this product within the targeted tissue, but also its dissemination into non-targeted tissues and biological fluids.

A large panel of assays to evaluate:

  • Gene therapy product biodistribution analysis in tissues and biological fluids by qPCR (absolute quantification of the transgenic genome)
  • Gene therapy product expression analysis in tissues by
    • RT-qPCR (relative quantification of the transgenic messenger),
    • Western-Blot and/or ELISA (specific detection of the transgenic protein).

PAC is also able to provide specific in vitro expression/potency assays for quality control release of a gene therapy product, after transduction of specific cells and relevant molecular/biochemical analysis.


Transblot
ELISA

An experimented core

The team has participated to a large number of preclinical studies in rodent, canine and nonhuman primate models. The core was also involved in toxicological gene therapy studies for muscle diseases. With this experience, PAC is also able to provide global coordination of in vivo experiments.

All preclinical analytics services are running under an ISO 9001, version 2015, approved quality system. All studies are performed to guarantee data and sample traceability. Validated procedures and approved documents are used for all types of analysis.

Equipment of the core PAC include :

  • a tissue lyzer,
  • an automated liquid handling workstation,
  • a nanophotometer,
  • PCR and qPCR machines,
  • a microplate washer,
  • an ELISA microplate reader
  • and a turbo gene transfer system.
qPCR
Hamiltion

The core is supervised by Dr Caroline Le Guiner, PhD and has dedicated human resources (1 full time technician and 2 part time engineers).