Transgenics
Genetically engineered rodent models provide an opportunity to investigate disease-relevant biology in a controlled, longitudinal setting. Transpharmation's Transgenics Platform combines established mouse and rat models with behavioral assessment, biomarker analysis, EEG and histology to help characterize disease phenotypes and evaluate therapeutic interventions across multiple neurological indications. The platform also offers the opportunity to work with additional transgenic models and tailor endpoints to be fit for purpose based on specific research objectives.
Detail of offering
Transpharmation has experience supporting studies across a diverse portfolio of transgenic and genetically modified rodent models. Rather than focusing on a single disease area, the platform is designed to provide flexible access to models that reproduce specific genetic drivers, pathological mechanisms or functional deficits relevant to therapeutic development. The flexibility of the platform extends to behavioral, biochemical and electrophysiological domains and customizing their application to a variety of transgenic models.
The platform includes both mouse and rat SOD1 G93A models, which develop progressive neurological deficits over time. These models are suited to longitudinal efficacy studies and can be combined with clinical observations, motor function assessments and additional endpoint packages to monitor disease progression and treatment response.
The ARTE10 APP/PS1 model provides a transgenic system for investigating progressive amyloid pathology. The model supports behavioral, biomarker and EEG assessments across multiple disease stages, enabling the evaluation of pathological progression alongside functional changes. Endpoints can include anxiety, cognition, agitation, sensorimotor gating, sleep parameters and fluid biomarkers such as neurofilament light chain.
Transpharmation has experience with multiple tau-based models, including P301L and GEM1-3 lines. These models enable investigation of tau-related pathology through behavioral assessment and molecular biomarker analysis, including measurements of total tau, phosphorylated tau and markers of neurodegeneration.
The platform includes knockout models targeting disease-associated genes, including PINK1 and PARK7 rat lines. These models provide opportunities to investigate the consequences of specific genetic alterations and to monitor functional changes over extended study periods.
Beyond neurodegenerative disease, the platform has experience supporting studies in several genetically modified models, such as models related to DISC1, SCN1A, Ts65Dn trisomy and C9orf72. These models can be paired with disease-relevant behavioral, biomarker and electrophysiological endpoints depending on study objectives.
Integrated Endpoint Strategy
All transgenic models can be characterized through a flexible combination of:
- Behavioral assessments
- Tissue and fluid biomarkers
- Wireless EEG and sleep analysis
- Histological assessment
This integrated approach enables both comprehensive phenotyping and the evaluation of treatment effects against established disease-relevant endpoints.
Applicable Portfolio Services
Have a program in mind?
Whether you are evaluating a novel target, characterizing a genetic disease mechanism or designing an efficacy study, our team can help identify the most appropriate transgenic model and endpoint strategy for your program.