This technology, FixR, is a modified peptide and delivery system aimed at reducing pathogenic late sodium currents, which are implicated in cardiac and neurological diseases, including cardiac arrhythmia.
A “late” or “persistent” sodium current disrupts sodium channel inactivation in the heart and results in cardiac arrhythmias and sudden cardiac death. Current therapeutics to regulate late sodium currents include small molecules with varying efficacy and low selectivity in targeting late currents over other currents present during the cardiac cycle, which can lead to frequent negative off-target effects. Therefore, identifying improved strategies with better selectivity for late sodium current inhibition is critical for developing potential therapeutics to treat cardiac arrhythmogenic syndromes.
This technology, called FixR, incorporates a peptide derived from the endogenous sodium channel modulator, fibroblast growth factor homologous factor (FHF), with a combined delivery system. The FHF derivative in FixR is a more potent inhibitor of late sodium currents than endogenous cardiac FHF, making it an ideal therapeutic. The delivery is conducted via adeno-associated virus (AAV) vectors and cell-penetrating peptides (CPP) for cell-specific delivery and control. While the technology is primarily aimed at cardiac disease, late sodium currents have a pathophysiological impact in neurological and pain-related diseases, creating the potential for FixR to be expanded as a therapeutic for non-cardiac diseases.
This technology has been validated in HEK293 cells, cardiomyocytes derived from patient-derived induced pluripotent stem cells (iPSCs), and myocytes from transgenic mice.
Patent Pending (US20250049953)
IR CU22077
Licensing Contact: Kristin Neuman
