The goal of this project is to find a cure for inherited arrhythmogenic cardiomyopathy (ACM), a rare cardiac disease. Therefore, researchers 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).
The research group of Van der Meer will investigate new therapies on pieces of heart tissue. They engineer heart tissue from patients with the use of pluripotent stem cells. Van der Meer: ‘We will reprogram skin cells to pluripotent stem cells, which we will then grow into heart muscle cells to make patient-specific heart tissue. We will test various new therapies on these pieces of heart tissue, including the use of gene therapy.’
The project brings together all expertise required to transform GEREMY into a translational success. Experts in clinical, preclinical research and cardiac genetics (Pieter Doevendans – NLHI/UMCUtrecht, Eva van Rooij – Hubrecht Institute/ UMCUtrecht, Peter van der Meer – UMCGroningen, 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 Italy, UEF Finland, NLHI Netherlands) will prepare for 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 that the project is continuously aligned with patient needs and ethical perspectives.
