Stoke Therapeutics Introduced Breakthrough Zorevunersen for the Treatment of Dravet Syndrome
Stoke Therapeutics has introduced zorevunersen, a groundbreaking treatment for Dravet syndrome, targeting cases with confirmed SCN1A gene mutations that are not associated with gain-of-function.
Zorevunersen is being developed as a potential first-in-class disease-modifying therapy for Dravet syndrome. This investigational antisense oligonucleotide (ASO) is currently undergoing evaluation in clinical trials.
Stoke believes zorevunersen can address the disease's genetic cause by enhancing NaV1.1 protein expression through the wild-type SCN1A gene, aiming to reduce seizures and related comorbidities.
Dravet syndrome is a severe, progressive genetic epilepsy that begins within the first year of life and is characterised by frequent, prolonged, and refractory seizures. The condition is notoriously difficult to treat and is associated with a poor long-term prognosis.
Beyond seizures, Dravet syndrome significantly impacts quality of life, with complications such as intellectual disability, developmental delays, movement and balance issues, speech and language disturbances, growth defects, sleep disorders, autonomic nervous system disruptions, and mood disorders.
Classified as a developmental and epileptic encephalopathy, the disease is marked by both cognitive impairment and developmental delays.
U.S. Food and Drug Administration has granted zorevunersen for the treatment of Dravet syndrome with a confirmed mutation.
