Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, provides an overview the company’s TANGO (Targeted Augmentation of Nuclear Gene Output) technology.
TANGO allows for precisely modulating splicing to increase a target protein’s expression by exploiting naturally occurring non-productive splicing events. Stoke is planning on using this technology to bring to clinical trials a potential therapy for Dravet syndrome.
Dravet syndrome, a severe form of epilepsy that is part of a group of diseases known as SCN1A-related seizure disorders.
Dravet syndrome usually appears during the first year of life as frequent fever-related (febrile) seizures. As the condition progresses, other types of seizures typically occur, including myoclonus and status epilepticus. A family history of either epilepsy or febrile seizures exists in 15% to 25% of cases. Intellectual development begins to deteriorate around age 2, and affected individuals often have a lack of coordination, poor development of language, hyperactivity, and difficulty relating to others.