zorevunersen

Zorevunersen is an investigational antisense oligonucleotide (ASO) developed for the treatment of Dravet syndrome, a rare and severe genetic epilepsy. It is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene, thereby restoring physiological NaV1.1 levels in patients with Dravet syndrome who have mutations in this gene[1][2][3][4][5]. This mechanism aims to reduce both seizure frequency and significant non-seizure comorbidities such as cognitive and behavioral impairments[3][4]. Zorevunersen has demonstrated substantial reductions in seizures and improvements in cognition and behavior during clinical trials, supporting its potential as a first-in-class disease-modifying therapy for Dravet syndrome[3][6]. The drug has received orphan drug designation from both the FDA and EMA, rare pediatric disease designation from the FDA, and Breakthrough Therapy Designation for Dravet syndrome[4][5].

Other names

zorevunersenSTK-001STK001STK 001