Human iPSC-Derived Cardiomyocyte Models for FRDA and DMD Therapeutic Assessment

This poster presents Ncardia’s human iPSC-derived cardiomyocyte platform for modeling Friedreich’s Ataxia (FRDA) and Duchenne Muscular Dystrophy (DMD), enabling physiologically relevant disease modeling and therapeutic evaluation in a scalable in vitro system. 

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• How patient-derived iPSC-cardiomyocytes recapitulate key disease phenotypes, including reduced frataxin levels in FRDA and absence of dystrophin in DMD 
  
• Quantitative functional readouts such as calcium handling abnormalities, ROS levels, and contractility changes reflecting disease pathology 
• How large-scale bioreactor-based manufacturing enables consistent, high-purity cardiomyocyte production for screening applications 
• How integrating patient-derived models into screening workflows supports more predictive therapeutic selection compared to traditional systems 

• Evidence of phenotype rescue through AAV-mediated gene therapy, restoring key functional and molecular markers in both FRDA and DMD models