NIH Grant Awarded ($670 K) to further Development of Drug to Treat Dravet Syndrome

LifeSplice was awarded an NIH Phase I/Phase II SBIR Fast-Track CREATE grant. The $670K Phase I grant focuses on additional pre-clinical development of LSP-SCN8. Video-EEG and electrophysiological characterization of LSP-SCN8A will be performed in preparation for follow on pre-clinical toxicology and pharmacodynamics testing in the Phase II portion of the grant. LSP-SCN8 induces a potent and specific reduction in fully functional SCN8A expression and has dramatically reduced or completely prevented seizures and mortality in a mouse model of Dravet Syndrome.