Project description DEENESFRITPL RNA therapeutics for cardiometabolic diseases Cardiometabolic diseases include diabetes, non-alcoholic fatty liver disease and atherosclerotic cardiovascular disease. Although these diseases frequently co-exist in patients, interventions have yet to be designed against their common targets and shared pathways. The EU-funded LiverTarget project brings together worldwide experts in the field to develop and test innovative therapies against such cardiometabolic diseases. Researchers will focus on ribonucleic acid (RNA) therapeutics such as small interfering RNA to silence the expression of inflammatory genes in the liver. These RNA molecules will be complexed with lipid nanoparticles for effective delivery to the liver, tested in preclinical mouse models for efficacy, and the lead candidates evaluated. Show the project objective Hide the project objective Objective 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.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.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: 1. Generate innovative siRNA pools and synthetic mRNAs to regulate ceramide and inflammatory targets in the liver. 2. Formulate them in established human-approved liver-targeting lipid nanoparticles (LNP). 3. Determine their efficacy in vivo in preclinical mouse models of obesity, diabetes, non-alcoholic fatty liver disease (NAFLD) and atherosclerosis.4. 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). This will result in a set of novel RNA-LNP formulations, directed against targets shared between ASCVD, diabetes and NAFLD, with demonstrated efficacy and toxicology, ready for further clinical development. It will also open up new directions of research towards the development innovative RNA therapeutics against liver targets for the treatment of cardiometabolic diseases, ultimately benefiting the patient, the society and the economy. Fields of science medical and health sciencesclinical medicineendocrinologydiabetesmedical and health sciencesclinical medicinehepatologynatural sciencesbiological sciencesgeneticsRNAmedical and health sciencesbasic medicinetoxicologymedical and health scienceshealth sciencesnutritionobesity Keywords siRNA nanomedicine ceramides cardiovascular disease diabetes NASH Programme(s) HORIZON.2.1 - Health Main Programme HORIZON.2.1.5 - Tools, Technologies and Digital Solutions for Health and Care, including personalised medicine Topic(s) HORIZON-HLTH-2021-TOOL-06-02 - Next generation advanced therapies to treat highly prevalent and high burden diseases with unmet medical needs Call for proposal HORIZON-HLTH-2021-TOOL-06 See other projects for this call Funding Scheme HORIZON-RIA - HORIZON Research and Innovation Actions Coordinator TAMPEREEN KORKEAKOULUSAATIO SR Net EU contribution € 2 018 750,00 Address KALEVANTIE 4 33100 Tampere Finland See on map Region Manner-Suomi Länsi-Suomi Pirkanmaa Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 2 018 750,00 Participants (8) Sort alphabetically Sort by Net EU contribution Expand all Collapse all UNIVERSITA DEGLI STUDI DI MILANO Italy Net EU contribution € 650 000,00 Address Via Festa Del Perdono 7 20122 Milano See on map Region Nord-Ovest Lombardia Milano Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 650 000,00 SITOOLS BIOTECH GMBH Germany Net EU contribution € 525 000,00 Address Lochhamerstrasse 29A 82152 Planegg See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Bayern Oberbayern München, Landkreis Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 525 000,00 SYDDANSK UNIVERSITET Denmark Net EU contribution € 400 000,00 Address CAMPUSVEJ 55 5230 Odense M See on map Region Danmark Syddanmark Fyn Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 400 000,00 IDRYMA IATROVIOLOGIKON EREUNON AKADEMIAS ATHINON Greece Net EU contribution € 618 750,00 Address SORANOU EFESIOU 4 115 27 Athina See on map Region Αττική Aττική Κεντρικός Τομέας Αθηνών Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 618 750,00 LAMDA THERAPEUTICS IKE Greece Net EU contribution € 262 500,00 Address SYGGROU AVENUE 190 176 71 ATHENS See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Αττική Aττική Νότιος Τομέας Αθηνών Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 262 500,00 UNIVERSITAIR MEDISCH CENTRUM UTRECHT Netherlands Net EU contribution € 400 000,00 Address HEIDELBERGLAAN 100 3584 CX Utrecht See on map Region West-Nederland Utrecht Utrecht Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 400 000,00 ASTRAZENECA AB Sweden Net EU contribution € 50 000,00 Address Vastra Maelarhamnen 151 85 Sodertaelje See on map Region Östra Sverige Stockholm Stockholms län Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 50 000,00 VECTOR B2B DRUG DEVELOPING ASSOCIACAO PARA INVESTIGACAO EM BIOTECNOLOGIA Portugal Net EU contribution € 1 075 000,00 Address AV. PROFESSOR EGAS MONIZ, EDIF. EGA MONIZ, PISO 2, SALA A8 1649-028 LISBOA See on map Region Continente Área Metropolitana de Lisboa Área Metropolitana de Lisboa Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 1 075 000,00