EUROSTARS CARDIOMYO consortium has launched a HTS in vitro drug screening platform to identify new therapies for cardiomyopathies in human iPSC cardiomyocytes

Leading scientists from Amsterdam University, Taros Chemicals and Zeclinics have combined expertise and state of the art technologies, to develop an innovative drug discovery platform and accelerate the discovery of new therapies in the field of cardiomyopathies and heart failure.

The project was funded by the prestigious EUREKA-EUROSTARS innovation program to respond to unmet medical needs for directed therapies in the field of cardiomyopathies. Diderick Kuster and Carol Ann Remme from Amsterdam University have successfully setup an in vitro drug screening platform and generated new models of hypertrophic and dilated cardiomyopathy in human iPSC cardiomyocytes. Specific mutations in the troponin gene are known to be associated with cardiomyopathies and the introduction of point mutations in this gene, with CRISPR-Cas9 based genome editing technology, allowed to mimics human clinical HCM and DCM phenotypes such as severe defects in cardiac function and contraction-relaxation in vitro.

The in vitro cardiomyopathy drug screening platform was launched in June 2022 and large-scale screening of compounds will:

  • Narrow down the large list of hundreds of compounds to tens of potential candidates
  • Identify compounds that can revert cardiac dysfunction associated with HCM and/or DCM
  • Shortlist the best candidates for in vivo phenotypic screening in zebrafish
  • Help, together with the in vivo screening, to select the best molecules to be targeted for medicinal chemical modifications and optimizations cycles to gain efficacy and prevent toxicity

The development and launching of the hiPSC cardiomyocyte drug screening platform by the CARDIOMYO consortium, sets the first milestones in the creation of new solutions to fight drug attrition in the field of cardiomyopathies.