Europe’s research and innovation funding programme Horizon2020 has granted research teams 8M euro funding for developing new effective therapies for rare diseases. Exom Group is proud to be a member of this consortium, called GEREMY. This project aims to find a cure for inherited arrhythmogenic cardiomyopathy (ACM), a rare cardiac disease. The GEREMY consortium will engineer disease models for proper assessment of therapeutic interventions and aims to provide in vitro and in vivo, preclinical proof-of-concept for various promising gene therapy approaches (oligonucleotide chemistries, gene editing and gene modulation).
Based on previous successes, GEREMY will target PLN & PKP2 as a roadmap of the technology. Restoring the primary defect in the PLN & PKP2 genes that cause the disease will lead to preserving or even restoring myocardial contractility. Through this approach, GEREMY will work towards the curative treatment of inherited ACM and likely other cardiomyopathies, which significantly burden the healthcare system.
The principal applicant for the GEREMY project is Pieter Doevendans (Netherlands Heart Institute: NLHI): “This grant means a lot to me as a clinical scientist. It will literally take our research field to the next level: from studying disease in a dish to assessing therapeutic efficacy in relevant preclinical models and determining patients’ needs. We will work to develop not only a treatment to reduce symptoms but try to develop a cure for this rare cardiac disease. We are proud and grateful for the support of the dutch PLN Foundation supporting the formation of the consortium.”
The project combines all expertise required to transform GEREMY into a translational success. Experts in clinical, preclinical research and cardiac genetics (Pieter Doevendans – NLHI/UMC Utrecht, Eva van Rooij – Hubrecht Institute/ UMC Utrecht, Peter van der Meer – UMC Groningen, Christian Kupatt – University Munich Germany, Seppo Yla-Herttuala – University of Eastern Finland, Mauro Giacca – King’s College London UK) will deliver preclinical efficacy and safety data for the mutation correction. The partners with regulatory and clinical trial expertise (EXOM GROUP Italy, UEF Finland, NLHI Netherlands) will prepare for the immediate start of clinical trials upon project completion and apply for orphan drug designation. The European Patients’ Academy on Therapeutic Innovation (EUPATI, https://eupati.eu/), a patient training organisation, and KUL (Leuven Belgium) ethics partner will ensure the project is continuously aligned with patient needs and ethical perspectives.