The TIGEM Translational Incubator was set-up in order to develop large-scale in vivo preclinical studies for the treatment of different inherited disorders by using standardized and optimized protocols to facilitate translation from the bench to the bedside. It is made up of dedicated scientists who perform pre- clinical research to fill gap between discovery and a clinical trial.
The Translational Incubator provides a wide range of preclinical testing services:
- in vivo administration of nanomolecules/compounds in vivo and ex vivo gene therapy approaches
- in wild type and inherited diseases animal models (small and large animal models) by using different administration routes: oral, intravenous, intraperitoneal, Intra CSF (intraventricular, lumbar, intra cisterna magna) subcutaneous and local administration.
The Facility collaborates with both TIGEM investigators and/or with external collaborators (academic and pharmaceutical companies) to develop experimental design, formulation, administration and assessments of potential therapeutic compounds, as well as the interpretation of study results. Approaches include
- Standardized experimental procedures;
- Assay set-up
- Pre-clinical studies in diverse animal models, doses and timing
- Planning and overview of IND-enabling non clinical studies
- Animal house for small and large animals
- Availability of different mouse models of LSDs (MSD, MPSs and others)
- Formulation and administration of compounds
- Production of viral vectors
- Tissue collection
- Biochemical and IHC analysis Interpretation of results and statistics