Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company focused on developing innovative treatments for severe genetic diseases. The company specializes in addressing haploinsufficiencies by enhancing the expression of healthy proteins rather than directly modifying genes.
Zorevunersen is an investigational medicine currently undergoing phase I/II clinical trials aimed at treating Dravet syndrome, a severe form of genetic epilepsy characterized by frequent seizures and developmental delays.
By upregulating protein expression, zorevunersen aims to restore functional protein levels, ultimately alleviating the symptoms associated with Dravet syndrome.
These patients face challenges due to a significant loss of protein function leading to severe developmental and physical disabilities.