Blog, News and Events

11 September 2025

Cutting Edge Gene Editing in Primary Human Hepatocytes

Gene editing promises cures for a wide range of diseases. Zinc-finger nucleases (ZFNs) were developed as the first programmable editors in the early 2000s, followed by TALENs (Transcription Activator Like Effector Nucleases) about a decade later[1]. However, it was the discovery and application of easy to use CRISPR/Cas9 gene editing methods, that sparked a flurry of new activity since 2013. New gene editing methods that build on this platform continue to be a hot area of research, with the goal of developing techniques that increase specificity and circumvent the need for double-stranded breaks (DBSs) in order to reduce off-target effects and minimize safety concerns[2]. Moreover, increasing the size of sequences that can be modified, and improving editing efficiency in non-dividing cells are also areas of great interest.

Blog, PXB-cells

19 August 2025

Advancing the Development of Safe & Effective LNP-Delivered Therapies using Humanized Liver Models

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.

PXB-mouse, Blog

18 August 2025

Bridging the translational gap: Humanized Liver models as predictive tools for RNA therapeutic success

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].

PXB-mouse, Blog

17 June 2025

From modeling disease to supporting translational preclinical studies: a year in humanized liver model research

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.

Blog

19 December 2024

Humanized Liver Models: Revolutionizing RNA Therapeutic Development

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.

PXB-mouse, News, Blog

9 December 2024

Targeting cccDNA in Human Hepatocytes: Pathway to a Cure for HBV?

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.

Blog, PXB-cells

2 May 2024

Increasing translational potential with PXB-mice as an alternative to non-human primates

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.

PXB-mouse, Blog

18 October 2023

Unlocking the Future of AAV-Based Gene Therapies

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.

News, Blog