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.


 

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:

1f
Generate innovative siRNA pools and synthetic mRNAs to regulate ceramide and inflammatory targets in the liver.
2f
Formulate them in established human-approved liver-targeting lipid nanoparticles (LNP).
3f
Determine their efficacy in vivo in preclinical mouse models of obesity, diabetes, non-alcoholic fatty liver disease (NAFLD) and atherosclerosis.
4f
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).


THE PROJECT

 

LiverTarget is an EU-funded Research and Innovation Action project designed to develop a new generation of advanced RNA therapeutics that will reduce disease burden e.g. myocardial infarct and will improve their quality of life in the future, if the lead compound(s) successfully pass the phase 1-3 clinical trials after this project.
LiverTarget will significantly boost European innovation capacity in the field of personalized nanomedicines using unique RNA technology. We expect that this innovative approach will later lead to beneficial health economic implications as it may lower the costs per quality- adjusted life year.



ARTIFICIAL
INTELLIGENCE

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DATA
SCIENCE

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VOICE ASSISTANTS

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MACHINE
LEARNING

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