Gene therapies

The growing research in the field of gene therapy and advanced therapies is leading to a dynamic development of the biopharmaceutical companies’ pipelines.

Large animal and particularly non-human primates (NHP) are key animal models for the late preclinical testing of novel gene therapies (GTMPs) and other advanced therapy medicinal products (ATMP).

Cynbiose makes available both, its expertise on this valuable in vivo model as well as its AAALAC- accredited facility that has all the required authorizations for dealing with GMOs up to Biosecurity level 2 (barrier housing, waste handling, …).

We provide a dedicated solution to assess viral and non-viral gene therapy candidates (AAV, adenovirus, oncolytic virus, ….) with be-spoke non-clinical studies and high quality services in a GLP-like quality management system.

Non-clinical assessment of gene therapies & oncolytic viruses

Preclinical studies

Our services aim at assessing the pharmacology and safety of viral and non-viral gene therapy medicinal products:

• Evaluation of the pharmacokinetics, pharmacodynamics and Biodistribution
• Evaluation of the immunogenicity
• Safety and Toxicology assessment
• Screening of capsid libraries
• Efficacy studies on NHP physiopathological CNS models
• Design of flexible protocols

These studies support the preclinical evaluation of drug candidates developed in different therapeutic areas : central nervous system, oncology, genetic disorders, hematological  disorders, musculoskeletal disorders, …

In-life studies

• Prior to conducting non-clinical studies, and if required, a screening can be performed to select seronegative animals (cellular assays).
• Delivery routes : intravenous (IV), intrathecal (IT), intracerebral (ICV, ICM), ….
• In vivo non-invasive imaging (MRI)
• Clinical monitoring
• Chronic or one-off collection of fluids (including CSF), tissues & organs
• Bioanalysis
  • Evaluation of the vector biodistribution/shedding and the expression profiles (qPCR)
  • Evaluation of product expression in tissues (RT-qPCR, Western blot, ELISA)
  • Immuno-monitoring
  • Toxicity: immunotoxicity, hepatotoxicity, neurotoxicity
  • Histopathology
  • Other assays available on request

For more information on our capabilities and services, please contact us.