in vivo Pharmacology Studies for CGT
CGT in vivo Pharmacology Studies for Preclinical Programs
Pharmaron offers specialized CGT in vivo pharmacology support to accelerate cell and gene therapy development. Leveraging advanced models and imaging technologies such as IVIS, our studies provide critical insights into drug distribution and persistence, cell fate dynamics, viral shedding, transgene and biomarker expression, as well as disease and tumor model evaluation. These capabilities enable a deeper understanding of therapeutic activity, safety, and translational potential—supporting confident decision-making throughout the development process.
Capabilities
Drug Distribution, Persistence & Viral Shedding
Biodistribution of a gene therapy product helps characterize the level and persistence of the viral vector and its therapeutic transgene. This is a critical first step to support the design and duration of safety studies as well as link expression levels to safety and efficacy results. Viral shedding evaluates the dissemination of a viral vector from the host body and serves as a measure of potential risk to third parties or the environment. Pharmaron can develop de novo assays utilizing state-of-the-art PCR technology to support the analysis and validate them as needed.
Cell Fate Dynamics
One of the primary endpoints for a preclinical cell therapy study includes the cell therapy product fate post-administration. This includes survival, engraftment, distribution, differentiation, and integration of the therapeutic cells. Assessment of cell fate contributes to characterization of the product’s activity and safety profile, while helping to justify animal selection, study duration, and potential target organs of toxicity. Pharmaron’s integrated bioanalytical platforms allow for assessment of cell fate through flow cytometry, PCR, MSD, IHC, and IVIS imaging.
Gene Expression and Biomarker Analysis
Pharmaron provides comprehensive gene expression and biomarker analysis to support cell and gene therapy development. Our platforms include PCR/ddPCR for nucleic acid quantification, ELISA for protein detection, and LC-MS for sensitive, multiplex biomarker profiling. These assays deliver precise insights into pharmacokinetics, drug exposure, and molecular response at both the DNA and protein level.
Disease and Tumor Models
Pharmaron provides a wide range of disease and tumor models, including ocular, diabetes, and CNS, to support the evaluation of cell and gene therapy programs. We also establish customized models based on sponsor portfolios or published literature to ensure therapeutic relevance. These models enable robust assessment of efficacy, safety, and mechanism of action, with advanced imaging and translational biomarkers delivering high-value data to guide development.
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