The rapid growth of RNA therapeutics, including siRNA and mRNA, is transforming the pharmaceutical and medical landscape, offering unprecedented potential for treating diseases ranging from viral infections to metabolic disorders. Despite this promise, translating these groundbreaking treatments from the lab to the clinic is fraught with challenges.
Traditional animal models, while invaluable for basic research, frequently fail to predict human responses due to species-specific differences in physiology, metabolism, and disease manifestation. These limitations often result in late-stage clinical trial failures, wasting significant time, effort, and resources.
In their article, “Bridging the Translational Gap: Humanized Liver Models as Predictive Tools for RNA Therapeutic Success”, Matthew Baginski and Dr. Sara Donnelly of PhoenixBio shed light on an innovative solution: the PXB-mouse. This humanized liver chimeric mouse model has been engineered to provide a more accurate and human-relevant platform for preclinical testing. Featuring livers populated with human hepatocytes, PXB-mice enable more precise assessment of drug efficacy, metabolism, and safety, addressing the critical translational gap in RNA therapeutic development.
The article presents several compelling case studies that illustrate the unique utility of the PXB-mouse. From improving siRNA delivery to reducing off-target effects and predicting clinical outcomes, these translational model are proving indispensable in developing next-generation therapies. Particularly valuable for RNA therapeutics like siRNAs, PXB-mice provide insights that are difficult to achieve with traditional models.
With over 400 peer-reviewed publications validating their value, PXB-mice also align with FDA guidelines for selecting preclinical tools for cell and gene therapies. By incorporating these advanced models into research pipelines, scientists can significantly improve the chances of clinical success, accelerating the availability of life-changing therapies for patients worldwide.
Explore the full article here to uncover how humanized liver mouse models are shaping the future of RNA therapeutics. Our team is happy to discuss this application and your research projects in more detail over email, web-conference, or onsite visit. Feel free to contact us at any time: