The MONARCH Study, conducted by Stoke Therapeutics, is currently enrolling for a Phase1/2a trial of STK-001. This new therapeutic medicine acts to specifically increase the number of healthy sodium channels (Nav1.1) that are impacted by mutations in SCN1A gene, the most common cause of Dravet syndrome. The study will focus on safety and tolerability, as well as assessing the efficacy of STK-001 to reduce seizure activity and improve patient health.
STK-001 is part of the TANGO approach that Stoke Therapeutics is leveraging to treat genetic haploinsufficiency. STK-001 is an antisense oligonucleotide that modifies the splicing of pre-mRNA from the healthy copy of the SCN1A gene, resulting in mRNA transcripts that produce more Nav1.1 sodium channels. Stoke recently published a paper detailing the mechanisms of how this technology works in cells and mice. Read the open-access article here: https://www.nature.com/articles/s41467-020-17093-9.
You can find more information under the Stoke MONARCH study tab at our website, including links to more information about the study, how to enroll, and a simplified explainer for the mechanism of action for STK-001. https://www.dravetfoundation.org/dsf-funded-research/participate-in-research/