Stoke Therapeutics Strengthens Board with Appointment of Dr. Clare Kahn
Stoke Therapeutics, Inc., a biotechnology company focused on transforming the treatment landscape for genetic diseases, has announced the appointment of Clare Kahn, Ph.D., to its Board of Directors. The addition of Dr. Kahn comes at a critical stage for the company as it advances its lead investigational therapy, zorevunersen, aimed at treating Dravet syndrome—a rare and severe neurological disorder.
Headquartered in Bedford, Massachusetts, Stoke Therapeutics is pioneering a novel approach to medicine by harnessing RNA-based technologies to restore protein expression. The company’s scientific strategy is centered on addressing the root cause of genetic diseases rather than merely managing symptoms, positioning it at the forefront of next-generation therapeutics.
Leadership Welcomes a Seasoned Industry Expert
Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics, expressed strong enthusiasm about Dr. Kahn’s appointment. He highlighted her extensive experience spanning more than 30 years in the pharmaceutical and biotechnology industries, particularly in regulatory strategy and drug development.
According to Smith, Dr. Kahn’s expertise is especially valuable as the company progresses its Phase 3 clinical study of zorevunersen. Her deep understanding of regulatory pathways and rare disease development is expected to strengthen the company’s strategic direction and accelerate its mission to deliver a disease-modifying therapy for patients with Dravet syndrome.
Smith emphasized that the addition of Dr. Kahn complements the existing leadership team and Board, enhancing Stoke’s ability to navigate complex clinical and regulatory milestones. Her insights are anticipated to have an immediate and meaningful impact as the company works toward bringing innovative treatments to patients with significant unmet medical needs.
Dr. Clare Kahn’s Extensive Background and Expertise
Dr. Clare Kahn brings a wealth of knowledge and leadership experience across multiple aspects of drug development, including early-stage research, clinical development, regulatory approval, and lifecycle management. Her career reflects a consistent track record of success in advancing therapies from concept to commercialization.
Most recently, Dr. Kahn served as R&D Strategy Officer and Chief Regulatory and Preclinical Development Officer at X-VAX Technology Inc., where she played a key role in shaping research strategies and overseeing regulatory and preclinical programs. Her leadership in these areas has contributed to the advancement of innovative therapeutic approaches.
Prior to her role at X-VAX, Dr. Kahn held positions of increasing responsibility at major pharmaceutical companies, including Pfizer and GlaxoSmithKline. During her tenure at these organizations, she gained extensive experience in global drug development and regulatory affairs, working on a broad range of therapeutic areas.
In addition to her executive roles, Dr. Kahn currently serves on the Board of Directors for Solid Biosciences and has provided advisory support to numerous early-stage and established biotechnology companies. Her ability to guide organizations through complex development and regulatory landscapes has made her a highly respected figure in the industry.
Dr. Kahn holds a Ph.D. in Biochemical Pharmacology from The Royal Postgraduate Medical School in London, further underscoring her strong scientific foundation.
Commitment to Advancing Dravet Syndrome Treatment
In response to her appointment, Dr. Kahn expressed excitement about joining Stoke Therapeutics and contributing to its mission. She noted the significant potential of zorevunersen to fundamentally alter the course of Dravet syndrome by targeting its underlying genetic cause.
Dr. Kahn highlighted the importance of developing therapies that go beyond symptom management and instead address the root mechanisms of disease. She conveyed her enthusiasm for supporting the company’s efforts to bring a transformative treatment to patients and families affected by this devastating condition.
Her perspective aligns closely with Stoke’s core philosophy of leveraging RNA medicine to restore normal protein levels, offering hope for long-term disease modification rather than temporary relief.
Understanding Dravet Syndrome: A Severe Neurological Disorder
Dravet syndrome is a rare and severe form of developmental and epileptic encephalopathy (DEE). It typically begins in infancy and is characterized by frequent, prolonged seizures that are often resistant to conventional anti-seizure medications.
The condition is most commonly caused by mutations in one copy of the SCN1A gene. This mutation leads to insufficient production of the NaV1.1 protein, which plays a crucial role in regulating neuronal activity in the brain. The resulting imbalance contributes to the hallmark seizures and neurological impairments associated with the disease.
Broad Impact on Patients and Families
Beyond seizures, Dravet syndrome significantly affects multiple aspects of development and daily functioning. Patients often experience cognitive impairment, developmental delays, and difficulties with movement and coordination. Additional challenges may include speech and language disorders, sleep disturbances, and behavioral issues.
The condition also impacts the autonomic nervous system, which controls essential bodily functions such as heart rate and digestion. These complications can contribute to a reduced quality of life for both patients and their caregivers.
Even with the best available treatments, many patients continue to experience uncontrolled seizures. Studies indicate that up to 57 percent of individuals with Dravet syndrome do not achieve a 50 percent reduction in seizure frequency despite treatment with existing anti-seizure medications.
Elevated Risks and Unmet Medical Needs
Dravet syndrome is associated with a significantly higher risk of sudden unexpected death in epilepsy (SUDEP) compared to other forms of epilepsy. Tragically, up to 20 percent of children and adolescents with the condition may not survive into adulthood due to complications such as prolonged seizures, seizure-related accidents, or infections.
The disease imposes a substantial emotional, physical, and financial burden on families, highlighting the urgent need for more effective treatment options.
Despite ongoing research and the availability of various anti-seizure therapies, there are currently no approved treatments that address the underlying genetic cause of Dravet syndrome. This gap underscores the importance of developing disease-modifying therapies like zorevunersen.
Global Prevalence and Impact
Dravet syndrome occurs worldwide and is not confined to any specific geographic region or ethnic group. It is estimated that approximately 38,000 individuals are living with the condition across major global markets, including the United States, the United Kingdom, the European Union, and Japan.
Given its rarity and complexity, Dravet syndrome remains an area of high unmet medical need within the broader field of neurology and genetic disorders.
Stoke Therapeutics’ Vision for the Future
Stoke Therapeutics is committed to advancing innovative RNA-based therapies that have the potential to transform the treatment of genetic diseases. By focusing on restoring protein expression, the company aims to address the root causes of disease rather than merely alleviating symptoms.
The development of zorevunersen represents a significant step forward in this mission. As a first-in-class investigational therapy, it holds the promise of becoming a disease-modifying treatment for Dravet syndrome—potentially changing the standard of care and improving outcomes for patients.
The appointment of Dr. Clare Kahn to the Board of Directors reflects Stoke’s dedication to strengthening its leadership and expertise as it moves closer to achieving this goal.
The addition of Dr. Clare Kahn to Stoke Therapeutics’ Board of Directors marks an important milestone for the company. Her extensive experience in drug development and regulatory strategy is expected to play a crucial role in guiding the company through its next phase of growth.
As Stoke continues to advance its innovative RNA-based therapies, the company remains focused on delivering meaningful solutions for patients with serious genetic conditions. With promising developments underway and strengthened leadership, Stoke Therapeutics is well-positioned to make a lasting impact in the field of biotechnology and rare disease treatment.
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.
