Stoke Therapeutics Announces First Patient Dosed in MONARCH Study in Dravet Syndrome


Stoke Therapeutics has announced that the first patient has been dosed with STK-001 in the phase 1/2a MONARCH study of children and adolescents ages 2 to 18 years old with Dravet syndrome. STK-001 has the potential to be the first disease-modifying medicine for Dravet syndrome, a severe and progressive genetic epilepsy that is characterized by developmental delays and cognitive impairment in addition to seizure activity.

“The start of MONARCH marks Stoke’s official transition to a clinical-stage biotech company,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “We enter this new stage in a strong financial position to execute on our plans for STK-001 in Dravet syndrome and continue to advance the potential of our TANGO platform for additional genetic diseases.”

Stoke Therapeutics was founded in 2014 by Apple Tree Partners to develop antisense oligonucleotide medicines to increase gene expression, to treat genetic epilepsies and other severe monogenic diseases. The company’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology platform has potential to treat a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50 percent of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Using the TANGO platform, Stoke aims to increase protein production from the remaining healthy copy of a gene and to restore the protein to near normal levels, thereby slowing or stopping disease progression. Beyond epilepsies, potential applications of Stoke’s TANGO technology include genetic conditions affecting the central nervous system, eye, liver, and kidney.

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