Livertarget – Innovative precision treatments

INTRODUCTION

THE
PROBLEM

Obesity, diabetes, non-alcoholic fatty liver disease (NAFLD) and atherosclerotic cardiovascular disease (ASCVD), in turn, are highly prevalent devastating diseases of our times despite available medicines, accounting for more than one in two deaths worldwide. They frequently co-exist, in the same patient, and constitute independent risk factors for each other; yet, unified approaches directed against common targets and shared mechanisms are lacking.

THE
SOLUTION

RNA Therapeutics comprise a new rapidly expanding category of drugs including small interfering RNAs (siRNA) and messenger RNAs (mRNA) that have attracted tremendous interest recently due to their first in-class drug approvals and notable success of the mRNA vaccines in the clinic.
LiverTarget aims to develop and validate novel RNA inhibition and activation therapeutics against cardiovascular and metabolic diseases by targeting ceramide synthesis and/or inflammatory cascades in the liver.

DATA SCIENCE

AIMS

LiverTarget aims at exploiting Partners years of research in the field of cardiometabolic research, the identification of ceramide pathways and inflammatory targets, and the development of novel siRNA technologies and lipid formulations, to:

Generate innovative siRNA pools and synthetic mRNAs to regulate ceramide and inflammatory targets in the liver.

Formulate them in established human-approved liver-targeting lipid nanoparticles (LNP).

Determine their efficacy in vivo in preclinical mouse models of obesity, diabetes, non-alcoholic fatty liver disease (NAFLD) and atherosclerosis.

Complete the necessary safety and toxicology evaluation of the most promising lead candidates and prepare the preclinical evaluation dossier for the European Medicines Agency (EMA).and atherosclerosis.