We are revolutionizing the translational potential of animal models in preclinical research. Discover the latest articles and news about our recent activities.
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.
7 August 2023
Hepatitis B is a serious and globally prevalent disease with 296 million people worldwide living with chronic hepatitis B virus (HBV) infections. Recent advances in HBV therapeutics have made infection a treatable disease, but they only disrupt certain areas of the HBV lifecycle. As a result, if the therapeutics are withdrawn, patients can relapse. What’s more, long-term use can cause antiviral resistance. To cure the disease, therapeutics need to address every stage of the HBV lifecycle, providing both seroclearance of the hepatitis B surface antigen (HBsAg) and silencing of the genomically integrated HBV DNA. However, investment in this space carries considerable risk as developers struggle to move treatments from animal models to human studies. Notably, human responses to HBV therapies are notoriously difficult to predict and traditional animal models translate poorly to the human environment, causing significant delays, as well as safety, cost, and efficacy concerns.
24 July 2023
How can humanized liver models provide accurate efficacy measurements at each stage of the viral lifecycle? Hepatitis B is a serious global health threat. Around 1.5 million hepatitis B virus (HBV) infections were identified in 2019 alone and around 296 million people are thought to be living with the chronic disease.¹ Researchers are now targeting previously unexplored areas of the viral lifecycle to get closer to developing a complete cure.
10 July 2023
In recent years, we have seen an explosion in the number of adeno-associated virus (AAV) vector-based gene therapies progressing to clinical trial stages, and subsequently an increasing number of regulatory successes. The reasons for this are clear. The versatility of AAV-based platforms makes them prime technology for targeting a range of tissues and pathologies, through their ability to silence, replace or modify genes.
