About Stoke Therapeutics
Stoke Therapeutics, Inc. is a biotechnology company. The Company focuses to treat the underlying causes of severe genetic diseases by upregulating protein expression. It is developing antisense oligonucleotide (ASO) medicines that target ribonucleic acid (RNA) and modulate precursor-messenger RNA (pre-mRNA) splicing to upregulate protein expression where needed and with appropriate specificity to near normal levels. It focuses to develop the precision medicine platform to target the underlying cause of a spectrum of genetic diseases in which the patient has healthy copy of a gene and mutated copy that fails to produce a protein essential to health. The Company utilizes its technology platform, Targeted Augmentation of Nuclear Gene Output (TANGO) to design ASOs to upregulate the expression of protein by individual genes in a patient. Its lead product candidate, STK-001, is offered to treat Dravet syndrome.