Our Approach

CardioReGenix capitalizes on recent discoveries in non-coding (nc)RNA-based therapeutic strategies, rational computational vector design and molecular engineering of AAV and Ad vectors by the CardioReGenix partners. CardioReGenix aims to develop a highly innovative human gene therapy approach using these emerging ‘next-generation’ ATMP platforms for the treatment of CVD. There are several advantages of ncRNAs (or their cognate inhibitors) as a therapeutic modality compared to more conventional protein-based approaches. Notable in this list is the class of circRNAs, the latest member of the non-coding RNAs. A growing body of evidence is emerging that they have regulatory functions and may play crucial roles during homeostasis and pathologies.

Typically, ncRNA can hit more than one pathway resulting in potential additive or synergistic effects. Some ncRNA may even be equivalent to a ‘molecular master-switch’, critically important in regulating cellular physiology in normal or pathologic conditions, particularly in the context of CHD and HF. As some ncRNAs (lncRNAs) naturally function at a relatively low copy number per cell, less than 100, therapeutic efficacy can be achieved with lower amounts of vector. Preliminary experiments conducted by the CardioReGenix partners revealed that some specific ncRNA are highly promising targets and have been shown to have beneficial effects in animal models of CHD and HF, justifying their exploration in clinical trials. Hence, ncRNAs represent attractive alternatives to protein-based approaches and therefore constitute the main focus of CardioReGenix.