Opis projektu
Leki na bazie RNA w profilaktyce niewydolności serca
Niewydolność serca jest często przyczyną zgonów oraz zachorowalności, dla której brakuje odpowiednich metod leczenia. Włóknienie serca to proces nadmiernej produkcji kolagenowej tkanki bliznowatej przez fibroblasty serca, który stanowi główny czynnik rozwoju niewydolności serca. Finansowany przez UE projekt FIBREX ma na celu opracowanie i doprowadzenie do gotowości klinicznej innowacyjnego, opartego na niekodującym RNA (ncRNA) produktu terapeutycznego na bazie oligonukleotydów antysensowych, przeznaczonego do leczenia niewydolności serca związanej z włóknieniem serca. Projekt bazuje na odkryciu, że najbardziej obiecującym celem jest imprintowany długi gen ncRNA MEG3, który ulega ekspresji w fibroblastach serca i rozregulowaniu w modelach niewydolności serca.
Cel
The aim of FIBREX is developing a novel, highly innovative and close to clinical readiness noncoding RNA (ncRNA)-based antisense oligonucleotide therapeutic for the treatment of heart failure (HF) derived from cardiac fibrosis. HF is the most common cause of morbidity and mortality with pressing social and economic burden. HF remains a deadly disease with no curative treatment, underlining the need for innovative therapeutic strategies. The revolutionary technology we are proposing acts by reversing cardiac fibrosis, the main driver of HF development and progression. The excess fibrotic tissue leads to continous stiffening and impairment of the heart muscle. In our prior work, ncRNA Meg3 was identified as the most promising target, expressed mainly by cardiac fibroblasts and by consistent transcriptional dysregulation in HF models. The critical role for Meg3 in cardiac fibrogenesis was validated both in vitro and in vivo and a lead compound targeting Meg3 showed promising efficacy in relevant models, human tissues and excellent exploratory safety data. We propose a development project for an antiMEG3 inhibitor to reach close to clinical readiness, by completing non-clinical pharmacodynamic and safety studies.This work will be carried out by an experienced team headed by Prof. Thum, with a track record of discovering and licensing various ncRNA inhibitors programs, and creating a clinical stage spin-off company Cardior, reaching phase 2 development and Series B funding. We are confident that our technology with the requested support will be further advanced into an innovative novel drug candidate and, driven by the substantial value increase, the project will be transitioned into a spin-off drug development or a licensing-deal with the industry. The development of the unique, RNA-based antiMEG3 therapeutic approach for HF derived from excessive fibrosis offers a new opportunity to revolutionize medical practice, reduce health care costs and improve patient's life.
Dziedzina nauki
Słowa kluczowe
Program(-y)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Zaproszenie do składania wniosków
HORIZON-EIC-2021-TRANSITIONOPEN-01
Zobacz inne projekty w ramach tego zaproszeniaSystem finansowania
HORIZON-EIC - HORIZON EIC GrantsKoordynator
30625 Hannover
Niemcy