BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. will present research at the upcoming annual meeting of the Oligonucleotide Therapeutics Society supporting its approach of leveraging antisense oligonucleotides to increase protein expression for the treatment of Dravet Syndrome and other monogenic diseases.
Isabel Aznarez, Ph.D., Stoke’s co-founder and vice president, head of biology, will present as part of Session VI of the conference, which is focused on target identification, drug discovery and trial design. Her presentation on using Stoke’s Targeted Augmentation of Nuclear Gene Output (TANGO) for the treatment of genetic diseases is scheduled for Oct. 2 from 12:05-12:20p.m. Pacific Standard Time.
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.
“Through our research, we’ve observed proof of target engagement and durable response in both wild-type and Dravet Syndrome model mice,” Aznarez said. “These findings demonstrate the efficacy of our TANGO platform as we’re advancing a differentiated pipeline of antisense oligonucleotide medicines.”
Visit the conference website to view the full agenda for the OTS Annual Meeting.
About Stoke Therapeutics
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 was launched in 2018 with a $40 million Series A investment funded by Apple Tree Partners. For more information visit www.stoketherapeutics.com and follow Stoke on Twitter @StokeTx.
