Selection of Relevant Animal Models
One of the key aspects of gene therapy product toxicology is the selection of appropriate animal models for preclinical studies. Our company employs a rigorous approach to this aspect, considering factors such as species-specific responses and relevance to human physiology. By selecting the most suitable animal models, we ensure the accuracy and reliability of our toxicology studies, enabling meaningful extrapolation to human patients.
Evaluation of In-Life Endpoints
In preclinical toxicology studies, the evaluation of in-life endpoints plays a crucial role in assessing the safety profile of gene therapy products. Our company employs a comprehensive set of in-life endpoints, including clinical observations, mortality, body weight, and clinical pathology, to monitor the physiological and pathological effects of gene therapy interventions. By meticulously analyzing these endpoints, we gain valuable insights into the potential risks and benefits of gene therapy products.
Immunogenicity Assessment
Assessing the immunogenicity of gene therapy products is essential for evaluating their safety and efficacy. Our company utilizes validated or qualified assays to evaluate immunogenic responses against both the gene therapy product and the expressed transgene. By examining the host immune response, we can identify potential immune-mediated adverse events and mitigate the risks associated with gene therapy interventions.
Comprehensive Study Design
In addition to gene therapy products, our company offers comprehensive toxicology testing services for cell therapy products. Our approach to study design is tailored to the unique attributes of each cell therapy product, ensuring the robustness and reliability of our safety assessment studies. Whether it involves ex vivo genetically modified cells or stem cells derived from various sources, we employ state-of-the-art methodologies to assess the safety and efficacy of cell therapy interventions.
Biodistribution Studies
Understanding the biodistribution of cell therapy products is essential for predicting their therapeutic effects and potential toxicity. Our company conducts biodistribution studies to determine the distribution of cell therapy products in vivo, including targeted and non-targeted tissues, biofluids, and the germline. By characterizing the biodistribution profile of cell therapy products, we can optimize treatment regimens and minimize the risk of off-target effects.
Viral Vector-Mediated Therapies
For gene therapy products delivered via viral vectors, our company offers specialized toxicology testing services tailored to the unique characteristics of viral vector-mediated therapies. Whether it involves adenoviruses, adeno-associated viruses, or herpes simplex viruses, we employ sophisticated techniques to assess key parameters such as viral shedding, infectivity, and persistence. By evaluating the safety profile of viral vector-mediated therapies, we facilitate the development of safe and effective gene therapy interventions.
