GEREMY

Gene Therapy for treatment of rare inherited Arrhythmogenic Cardiomyopathy 

The GEREMY consortium will use a novel parallel approach leveraging the transformative potential of gene therapy (GT) to deliver the first cardiac GT for treatment of rare inherited forms of ACM (desmosomal and non-desmosomal). Our approach also involves development of new preclinical models, validation of novel delivery vehicles and gene editing approaches, and the revolutionary development of pig models for in vivo GT validation.

Within the GEREMY project, we will develop GTs for PLN- and PKP2-mutation induced ACM. These will serve as models for other inherited heart diseases and contribute directly to the portfolio of treatments for rare cardiac diseases. Social embedding of all stakeholders will ensure value sensitive design and an innovative/modernised clinical trial design will contribute towards expedited timelines and increased probability of clinical development success. A business strategy will be established with commercialisation partners to market the selected GT approach; ultimately lowering mortality rates and reducing disease burden and healthcare costs.

EUPATI, as member of the Consortium, is in charge of ensuring a patient-centred approach, providing education and guidance to the researchers about patients engagement in clinical development, coordinate a multistakeholder advisory group and contribute to communication and dissemination activities.

Learn more about the project here: https://heart-institute.nl/research-portal/research-projects/geremy/