Preclinical Development

 

We support our clients in designing their studies in accordance with international guidelines and methods validation by developing intelligent designs for the testing that will meet the expectations of the authorities and that are designed the minimum required development for the forthcoming clinical testing.  We perform the studies in full compliance with GLP regulations and provide detailed study reports to support your regulatory submission.

The primary objective of your preclinical evaluation is to assess the toxicity and efficacy for your drug candidate providing you with accurate information of what can achieve your asset.

GenoSafe has a long and unique experience for carrying out the analytical testing of biological products with a special focus on novel therapies such as gene and cell therapies.

AAV, Lentivirus, Plasmid, CRISPR, CarT Cells

Main services

Biodistribution study

Gene expression analysis

Protein analysis

Immunogenicity assessment

Drug formulation evaluation

Germline transmission studies

 

Compounds

 

In vivo Gene Therapy

AAV vectors

Non integrative Lentivirus vectors

Non vectorised DNA (plasmid)

Ex vivo Gene Therapy (autologous or allogenic)

Lentivirus vectors

Retrovirus vectors

Car-T cells

CRISPER modifications

Plasmid

Somatic Cell therapy

Stem Cells (Differenciated)

Autologous modified cells

Tissue engineering

Monoclonal antibodies

Focus:  Biodistribution

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.