At a glance: ESGCT 33rd Annual Congress | 27–30 October 2026 | Congress Center Hamburg (CCH), Germany | Booth H-02
Liver-directed gene therapies often look promising in preclinical species, then behave differently in people. This month we're taking that problem to Hamburg. Here are three questions we're looking forward to discussing with gene and cell therapy teams at ESGCT.
Capsid tropism and transduction efficiency can vary sharply between species. The PXB-mouse® has a highly humanized liver, so you can compare capsids and measure transgene expression in human hepatocytes in vivo before committing to a lead.
For CRISPR, base editing and other gene-editing approaches, the PXB-mouse lets you assess editing efficiency in human hepatocytes in a living system. PXB-cells® give you a fresh human hepatocyte option for faster in vitro screening.
It can support early vector selection, dose-ranging, and translational packages ahead of the clinic. Our scientists can help you work out where it adds the most value in your program.
PXB-mice and PXB-cells have supported peer-reviewed research across gene therapy and editing modalities.
In vivo, in the PXB-mouse:
AAV-delivered CRISPR/Cas9 against HBV. AAV2-delivered CRISPR/Cas9 significantly suppressed HBsAg, HBcAg, HBV DNA and cccDNA in the livers of HBV-infected PXB-mice, with no significant off-target changes detected by whole-genome sequencing.
Development of an in vivo delivery system for CRISPR/Cas9-mediated targeting of hepatitis B virus cccDNA. Kayesh et al., Virus Res. 2020 Dec;290:198191. doi: 10.1016/j.virusres.2020.198191
LNP-delivered base editing against HBV. Two injections of an LNP-formulated adenine base editor achieved a ~90% reduction in serum HBV DNA in HBV-infected PXB-mice.
Adenine Base Editing Potently Suppresses Hepatitis B Surface Antigen Expression and Inhibits Hepatitis D Virus Release. Kumar et al., bioRxiv [Preprint]. 2026 Feb 6:2026.02.06.704371. doi: 10.64898/2026.02.06.704371.
In vitro, in PXB-cells:
Scarless, programmable edits. A retrotransposon-based editor achieved single-base changes, deletions and multiplexed insertions in quiescent PXB-cell hepatocytes.
Reprogramming site-specific retrotransposon activity to new DNA sites. Fell et al., Nature. 2025 Jun;642(8069):1080-1089. doi: 10.1038/s41586-025-08877-4.
Durable epigenetic silencing of PCSK9. An LNP-delivered epigenetic editor gave durable reduction of PCSK9 secretion in PXB-cells, outlasting a CRISPRi comparator. (Tremblay et al., Nature Medicine, 2025)
A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels. Tremblay et al., Nat Med. 2025 Apr;31(4):1329-1338. doi: 10.1038/s41591-025-03508-x.
Let's talk in Hamburg! Email na.li@phoenixbiousa.com to book a meeting, or stop by Booth H-02.