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.
Disruption of sodium channel inactivation leads to cardiac arrhythmias and sudden cardiac death. Current therapeutics for late sodium current control are small molecules which vary in efficacy and selectivity for late sodium currents over peak sodium currents. Additionally, these therapeutics frequently cause negative off-target effects through non-selective interactions with other ionic currents. Therefore, identifying improved strategies 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 an endogenous sodium channel modulator, fibroblast growth factor homologous factor (FHF) with a combined delivery system. These FHF proteins control late sodium currents and are the target of therapeutic intervention. The delivery is controlled by adeno-associated virus (AAV) vectors and cell penetrating peptides (CPP) that allow for cell-specific delivery and control. While the technology is primarily aimed at cardiac disease, late sodium currents have pathophysiological impact in neurological and pain-related 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(WO/2023/154933)
IR CU22077
Licensing Contact: Kristin Neuman