Stoke Therapeutics Announces Appointment of Dr. Clare Kahn to Its Board of Directors
Stoke Therapeutics, a clinical-stage biotechnology company focused on restoring protein expression through RNA-based medicines, has announced the appointment of Clare Kahn, Ph.D., to its Board of Directors. The addition of Dr. Kahn marks a strategic move for the company as it advances its lead investigational therapy, zorevunersen, which is being developed as a first-in-class, disease-modifying treatment for Dravet syndrome.
Stoke Therapeutics is at a pivotal stage in its corporate and clinical development trajectory. The company’s core mission centers on leveraging RNA medicine to address the underlying causes of genetic diseases by restoring normal protein levels. Its lead candidate, zorevunersen, is designed to target the root genetic deficiency associated with Dravet syndrome, a severe and lifelong form of epilepsy that begins in infancy and is characterized by frequent, prolonged seizures and significant developmental challenges. With the program advancing into late-stage clinical development, including a Phase 3 trial, the company is strengthening its leadership and governance to support this critical phase.
Commenting on the appointment, Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics, emphasized the importance of Dr. Kahn’s expertise at this juncture. He highlighted her more than 30 years of experience across the biopharmaceutical industry, particularly in regulatory strategy and drug development. According to Smith, Dr. Kahn’s background in advancing therapies for rare genetic diseases aligns closely with Stoke’s mission and will complement the capabilities of both the executive leadership team and the Board of Directors.
Smith noted that her insights are expected to play a meaningful role as the company progresses its Phase 3 study of zorevunersen. This late-stage trial represents a significant milestone for Stoke Therapeutics, as it aims to bring forward what could potentially become the first disease-modifying therapy for Dravet syndrome. If successful, such a therapy would move beyond symptomatic treatment and instead address the underlying genetic cause of the disorder, offering new hope to patients and their families.
Dr. Kahn brings an extensive and distinguished career in drug development, regulatory affairs, and strategic leadership. Over the course of her career, she has built a strong reputation for guiding therapies from early-stage research through clinical development, regulatory approval, and lifecycle management. Her experience spans both large pharmaceutical organizations and emerging biotechnology companies, providing her with a comprehensive perspective on the challenges and opportunities involved in bringing innovative therapies to market.
Most recently, Dr. Kahn served as R&D Strategy Officer and Chief Regulatory and Preclinical Development Officer at X-VAX Technology Inc., where she was responsible for shaping research and development strategy and overseeing regulatory and preclinical activities. Prior to that role, she held positions of increasing responsibility at major global pharmaceutical companies, including Pfizer and GlaxoSmithKline. During her tenure at these organizations, she contributed to the development and approval of multiple therapeutic products across a range of disease areas.
In addition to her executive leadership roles, Dr. Kahn currently serves on the Board of Directors of Solid Biosciences, a company focused on developing therapies for neuromuscular diseases. She has also provided advisory support to numerous early-stage and established biotechnology companies, further demonstrating her commitment to advancing innovation within the life sciences sector.
Dr. Kahn’s academic background further underscores her expertise in the field. She earned her Ph.D. in Biochemical Pharmacology from The Royal Postgraduate Medical School in London, where she developed a strong foundation in the scientific principles underlying drug development. This combination of scientific training and industry experience positions her to make valuable contributions to Stoke’s ongoing programs.
Expressing her enthusiasm for the new role, Dr. Kahn highlighted the transformative potential of Stoke’s approach to treating Dravet syndrome. She pointed to the company’s investigational medicine as a promising therapy that directly targets the underlying cause of the disease, rather than merely addressing its symptoms. According to Dr. Kahn, this approach has the potential to fundamentally change the treatment landscape for patients living with this debilitating condition.
She also conveyed her excitement about working with the Stoke Therapeutics team as they advance zorevunersen through clinical development. Dr. Kahn emphasized the importance of developing therapies that can make a meaningful difference in patients’ lives, particularly in rare genetic diseases where treatment options are often limited. She expressed confidence in the company’s scientific approach and its commitment to delivering innovative solutions for patients and families affected by Dravet syndrome.
The appointment of Dr. Kahn reflects Stoke Therapeutics’ broader strategy of strengthening its leadership team as it approaches key clinical and regulatory milestones. As the company continues to advance its pipeline and expand its capabilities, the addition of experienced industry leaders to its Board of Directors is expected to enhance its ability to navigate the complex regulatory landscape and bring novel therapies to market.
Overall, this development underscores Stoke Therapeutics’ commitment to advancing RNA-based medicines and addressing unmet medical needs in rare genetic diseases. With zorevunersen progressing through Phase 3 clinical trials and the support of seasoned leaders like Dr. Kahn, the company is positioning itself to potentially deliver a groundbreaking treatment that could significantly alter the course of Dravet syndrome and improve outcomes for patients worldwide.
About Dravet Syndrome
Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. Even when treated with the best available anti-seizure medicines (ASMs), up to 57 percent of patients with Dravet syndrome do not achieve ≥50 percent reduction in seizure frequency. Complications of the disease often contribute to a poor quality of life for patients and their caregivers.
Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP; up to 20 percent of children and adolescents with Dravet syndrome die before adulthood due to SUDEP, prolonged seizures, seizure-related accidents or infections1.
Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan2. There are no approved disease-modifying therapies for people living with Dravet syndrome.
About Zorevunersen
Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the unaffected (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition and behavior.
Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen (Nasdaq: BIIB) to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.
About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study.
Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts.
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