Biogen and Stoke Therapeutics have announced a collaboration to develop and commercialise zorevunersen, a potential first-in-class disease-modifying treatment for Dravet syndrome.
Zorevunersen has been granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) based on early clinical evidence suggesting significant improvements over existing treatments.
Zorevunersen is designed to address the root cause of Dravet syndrome by enhancing NaV1.1 protein production in brain cells using the non-mutated copy of the SCN1A gene.
Zorevunersen is an investigational antisense oligonucleotide (ASO) designed to target the SCN1A gene, which is responsible for most cases of Dravet syndrome.
Dravet syndrome is a severe genetic epilepsy disorder characterised by frequent seizures, cognitive impairment, and developmental delays.
Clinical studies of zorevunersen have shown reductions in seizure frequency and improvements in cognitive and behavioural functions in patients already receiving standard treatments, supporting its potential as a disease-modifying therapy.
Under the agreement, Biogen will handle commercialisation in all territories outside the United States, Canada, and Mexico, while Stoke will lead global development efforts.
The collaboration allows Biogen to expand its portfolio with a Phase 3-ready investigational treatment, leveraging its expertise in rare disease commercialisation.
The collaboration between Biogen and Stoke Therapeutics is expected to advance the development of zorevunersen, potentially offering a new treatment option for patients with Dravet syndrome worldwide.