PORTFOLIO

Our project is advancing a groundbreaking treatment for liver diseases that are characterised by impaired bile flow and tissue damage. This is an area of particularly high unmet medical need, as some of the liver diseases with bile flow stagnation have no approved medications. We have previously discovered a new target that shows great potential for a new drug therapy. With the support of the Gebert Rüf Innobooster, we will conduct critical proof-of-concept studies in long-term disease models, to validate the efficacy of our target and the developing lead candidate therapeutic.
Hence the Innobooster funding will accelerate our transition toward clinical trial readiness by generating essential preclinical data. Additionally, the support will enhance our business development activities such as expanding our advisory board with key opinion leaders and refining our regulatory strategy through consultations with regulatory offices. Altogether, the project support ensures our timeline towards clinical studies and market entry.

Prior to the start of the proof-of-concept (PoC), the optimal treatment conditions need to be defined. In a first step, the dosing of ClagalsiR will be determined with a response test. After the best dose is defined, we will check how long the desired targeting effects are stable. This allows then in a second step to set the optimal dosing frequency. With the optimal treatment conditions defined, the project can advance to the PoC study. In the PoC, the treatment efficacy will be validated in a in vivo model of established cholestatic liver disease, testing for liver injury blood-markers and histologic marker of cholestasis and fibrosis.