Stoke Therapeutics Inc
NASDAQ:STOK
Stoke Therapeutics Inc
Stoke Therapeutics, Inc. is a biotechnology company, which engages in the research and development of treatments for genetic diseases. The company is headquartered in Bedford, Massachusetts and currently employs 102 full-time employees. The company went IPO on 2019-06-19. The company focuses to treat the underlying causes of severe genetic diseases by upregulating protein expression. The company 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. The company 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 product candidate, STK-001, is offered to treat Dravet syndrome. Its research platform is designed to address protein deficiency.
Stoke Therapeutics, Inc. is a biotechnology company, which engages in the research and development of treatments for genetic diseases. The company is headquartered in Bedford, Massachusetts and currently employs 102 full-time employees. The company went IPO on 2019-06-19. The company focuses to treat the underlying causes of severe genetic diseases by upregulating protein expression. The company 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. The company 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 product candidate, STK-001, is offered to treat Dravet syndrome. Its research platform is designed to address protein deficiency.
Strong cash position: Stoke ended Q2 with $355 million in cash, expected to fund operations through mid-2028 and beyond the Phase III readout.
Phase III EMPEROR underway: Enrollment has started for the EMPEROR Phase III trial of zorevunersen in Dravet syndrome, with the first patient dosed and high interest from sites and families.
Promising long-term data: Three-year open-label extension data for zorevunersen showed sustained seizure reduction (59% to 91%) and continued improvements in cognition and behavior.
Favorable safety profile: Over four years and 700+ doses, zorevunersen has been generally well tolerated, with most adverse events mild and no new safety concerns.
Regulatory pathway: Zorevunersen holds breakthrough therapy designation; discussions with FDA for potential accelerated paths will take place in the second half of 2025.
Pipeline expansion: Phase I for STK-002 in Autosomal Dominant Optic Atrophy (ADOA) is now underway, supported by encouraging nonhuman primate data.
