Gene therapies

The growing research in the field of gene therapy and advanced therapies and the increasing number of players are leading to a dynamic development of the pipelines of biopharmaceutical companies

Non-human primates (NHP) are key animal models for the late preclinical testing of novel gene therapies (GTMPs) and other advanced therapy medical 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 gene therapies (AAV, adenovirus, , ….) with custom-made non-clinical studies and high quality services in a GLP-like quality management system.

Non-clinical assessment of gene therapies

Preclinical studies

Our services aim at assessing the pharmacology and safety (biodistribution, off targets, host immunity) of gene transfer therapeutic strategies:

• Pharmacodynamics
• Toxicology, biodistribution
• Efficacy studies on NHP physiopathological 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, cardiovascular, hematological  disorders, musculoskeletal disorders, cardiovascular diseases, infectious and respiratory diseases.

In-life studies

• Prior to conducting non-clinical studies and if required, a screening can be performed to select AAV seronegative animals with screening based on cellular assays.
• Delivery routes : intravenous, intramuscular, subcutaneous, oral, intrathecal, intracerebral, intra-articular, intraocular, inhalation
• Clinical monitoring
• Chronic or one-off collection of fluids (including CSF), tissues & organs
• In vivo non-invasive imaging (MRI)
• Bioanalysis
  • Evaluation of the vector biodistribution/shedding and the expression profiles (qPCR)
  • Evaluation of product expression in tissues (RT-qPCR, Western blot, ELISA)
  • Monitoring of host immunity : antibody response, ….
  • Monitoring of Cytokines:  Luminex, ELISpot, Simoa® for ultra-sensitive and translational biomarker detection
  • Histopathology
  • Other assays available on request

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