Stoke Therapeutics Inc. is a biotechnology company focused on developing innovative treatments for severe genetic diseases. The company was founded in 2014 by Dr. Adrian Krainer, Dr. Thomas Cooper, and Isabel Aznarez with the aim of addressing the underlying cause of genetic disorders by using RNA-targeted approaches.
Stoke Therapeutics is pioneering a novel therapeutic strategy called Targeted Augmentation of Nuclear Gene Output (TANGO). This approach involves using antisense oligonucleotides to increase the production of specific proteins from the non-mutated copy of a gene, compensating for the loss of function caused by a genetic mutation. The company's therapies are designed to treat a range of genetic disorders caused by haploinsufficiency, where the body does not produce enough of a particular protein due to a mutation in one copy of the gene.
Stoke Therapeutics has made significant progress in advancing its pipeline of potential therapies. Their lead investigational product candidate, STK-001, is being developed for the treatment of Dravet syndrome, a rare and severe form of epilepsy caused by a mutation in the SCN1A gene. In preclinical studies, STK-001 has shown promising results in restoring protein levels and reducing seizure activity in animal models.
Apart from Dravet syndrome, Stoke Therapeutics is actively exploring the application of their TANGO technology for other genetic diseases, such as genetic forms of Parkinson's disease and cystic fibrosis.
As a biotechnology company, Stoke Therapeutics collaborates with leading researchers and institutions in the field of genetics and molecular biology to advance their scientific understanding and accelerate the development of potential treatments. Their innovative approach has the potential to transform the lives of patients with severe genetic disorders and provide new hope for families affected by these conditions.