Biotechnology company focused on developing treatments for genetic diseases.
Stoke Therapeutics, Inc., headquartered in Bedford, Massachusetts, is a pioneering biopharmaceutical company focused on advancing innovative antisense oligonucleotide (ASO) therapies to address the root causes of severe genetic diseases. Leveraging its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) platform, Stoke Therapeutics designs ASOs tailored to selectively increase protein expression levels.
At the forefront of its pipeline is STK-001, the company's lead clinical candidate currently undergoing phase I/IIa trials. STK-001 aims to treat Dravet syndrome, a debilitating genetic epilepsy characterized by severe seizures. Additionally, Stoke Therapeutics is advancing STK-002 in the preclinical stage, targeting autosomal dominant optic atrophy, underscoring its commitment to addressing unmet medical needs in genetic disorders.
In pursuit of cutting-edge treatments, Stoke Therapeutics has forged strategic partnerships, including a significant collaboration with Acadia Pharmaceuticals Inc. This partnership focuses on developing RNA-based therapies for severe and rare genetic neurodevelopmental diseases affecting the central nervous system.
Founded in 2014 as ASOthera Pharmaceuticals, Inc., the company rebranded to Stoke Therapeutics, Inc. in May 2016 to reflect its expanded focus and dedication to transformative genetic medicines. With a steadfast commitment to innovation and patient-centric research, Stoke Therapeutics continues to pioneer advancements in the field of genetic disease treatment, aiming to improve outcomes and quality of life for patients worldwide.