The primary objective of a preclinical evaluation is to assess the safety of a drug candidate providing sufficient information for a proper risk assessment for the product’s use in human subjects.
GenoSafe has a long and unique experience in taking in charge non clinical studies for Gene and Cell-based therapeutic approaches.
We support our clients in designing their studies in accordance with international guidelines. We perform the studies in full compliance with GLP regulations and provide detailed study reports to support regulatory submission.
Our main services for preclinical evaluation include:
- Biodistribution study
- Gene expression analysis
- Protein analysis
- Immunogenicity assessment
The characterization of a product biodistribution profile following in vivo administration is a major component of a preclinical development program.
Biodistribution studies, required by regulatory agencies, are intended to determine the potential for a vector or a cellular agent presence and persistence in targeted tissues/biological fluids and their dissemination into non-target tissues/biological fluids and in the germline.
The biodistribution data, coupled with other preclinical safety endpoints help to determine whether product presence or gene expression correlates with any tissue-specific detrimental effects in the animals.
GenoSafe offers tissue/biological fluid analysis, using a quantitative PCR (qPCR) validated assay to determine the number of vector copies per microgram of genomic DNA.
In addition, the presence of a vector sequence in tissues/biological fluids may trigger further analysis to determine the transgene expression levels using reverse transcription quantitative PCR (RT-qPCR).
GenoSafe performs biodistribution studies in full compliance with GLP guidelines.