Stoke Therapeutics: Working on Rare Genetic Diseases скачать в хорошем качестве

Stoke Therapeutics: Working on Rare Genetic Diseases

Barry Ticho is the Chief Medical Officer at Stoke Therapeutics, and in this clip he provides an overview of his company. Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of severe genetic diseases, including genetic conditions affecting the central nervous system, eye and liver. Stoke’s lead program targets Dravet Syndrome, a rare form of genetic epilepsy that is characterized by severe and frequent seizures and cognitive impairment. By using antisense oligonucleotides to address haploinsufficiency of the sodium channel Nav 1.1., Stoke’s therapy aims to reverse the underlying cause of Dravet Syndrome, thereby preventing seizures and developmental decline. Aznarez will present preclinical data demonstrating an effective pharmacodynamic response to treatment in mouse models.