PORTFOLIO

This project advanced a new treatment approach for cholestatic, and fibrotic liver diseases characterized by impaired bile flow and progressive tissue damage, an area with high unmet medical need and limited or no approved therapies.

Building on our discovery of Claudin-3 as a novel target, the Gebert Rüf Innobooster enabled critical translational proof-of-concept work with our lead GalNAc-siRNA candidate, ClagalsiR.
The project was very successful and generated encouraging, decision-enabling preclinical data. We established an optimized GalNAc-siRNA dosing regimen and demonstrated claudin-3 target engagement and therapeutic effect in an established intrahepatic cholestasis model using clinically relevant readouts, helping to close an important translational gap toward further development.
These results directly supported the founding of CALDRE Ltd. in September, a Swiss biotech spin-out developing precise, liver-directed GalNAc-siRNAs to restore bile flow and prevent fibrosis. In parallel, the project strengthened our business development and visibility through a professional public presence, expanded advisory and KOL advisory board, and early partnering discussions at international biotech conferences.