We are revolutionizing the translational potential of animal models in preclinical research. Discover the latest articles and news about our recent activities.
19 August 2025
Oligonucleotide therapeutics have shown promise as a diverse treatment option for a wide range of diseases. Developing successful oligonucleotide therapeutics depends on being able to efficiently deliver the therapeutic to the target cells.
18 August 2025
The field of RNA therapeutics, with its potential for treating a wide range of diseases, continues to experience rapid growth and attracts significant investment. According to the American Society of Gene & Cell Therapy (ASGCT), as of Q1 2025, 35 RNA therapies have been approved globally and another 1,298 are currently in development (between preclinical and pre-registration stages) [1].
17 June 2025
PXB-mice and PXB-cells offer a highly physiologically relevant liver model for in vivo and in vitro research, respectively. They have been shown to have versatile applications in studies, ranging from hepatitis B virus (HBV) infection modeling to epigenetic research. In this round-up, we look back on the past year to showcase some of the most interesting and impactful research utilizing PXB-mice and/or PXB-cells, spanning disease modeling, drug metabolism, and epigenetic editing.
19 December 2024
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.
9 December 2024
Globally, around 250 million people are infected with hepatitis B virus (HBV) and more than 1 million more are estimated to become infected yearly. Researching the HBV viral life cycle and identifying potential novel therapeutics is challenging as only human and chimpanzee hepatocytes are permissive to HBV. However, due to the cost and ethical restrictions, alternative models are being explored. In vivo, rodent models including humanized liver chimeric mice (for example PXB-mice) and the AAV-HBV mouse model are being utilized. While these models are useful, each has their own pitfalls.
2 May 2024
Non-human primates (NHPs) share a wide range of genetic and physiological features in common with humans. Yet, despite their widespread use in preclinical research, NHP models suffer from several limitations that can lead to a lack of translatability in clinical trials.
18 October 2023
The promise of adeno-associated virus (AAV) vector-based gene therapies is undeniable. These therapies have already demonstrated potential in treating conditions like spinal muscular atrophy, retinal dystrophy, and hemophilia B, with the FDA approving three treatments as of early 2023. Yet, despite these successes, many promising therapies fail during clinical trials.
21 August 2023
It is estimated that 40% of drug candidates have failed to make it to market because of toxicity.¹ One of the most common adverse drug reactions is drug-induced liver injury (DILI), whereby patients experience acute illness, often with symptoms similar to hepatitis and cholestasis. Today, DILI is the leading cause of drug candidate failure and post-market withdrawals.² The high incidence of DILI in clinical trials, and even post-market events, is partly due to the use of conventional animal models at preclinical stages. Conventional models are notoriously poor predictors of efficacy and toxicity in the liver, due to species-to-species variation. In contrast, the PXB-mouse® model, with its humanized liver, provides a highly predictive model of human physiology and human-specific hepatotoxicity, allowing for more accurate prediction of human outcomes, and, therefore, aiding the smooth progression of new therapeutics into the clinic.
