Stoke Therapeutics Appoints Thomas McCauley as Chief Scientific Officer to Advance RNA Medicine Pipeline and Genetic Disease Research
Stoke Therapeutics, Inc. has announced the appointment of Thomas McCauley, Ph.D., as its new Chief Scientific Officer (CSO), reinforcing the company’s commitment to advancing innovative RNA-based medicines for severe genetic diseases. The appointment comes at a pivotal time for Stoke as it continues the late-stage clinical development of zorevunersen, its lead investigational therapy for Dravet syndrome, while expanding its proprietary RNA medicine platform into additional disease areas.
Dr. McCauley brings more than 25 years of biotechnology and pharmaceutical research and development experience, having successfully led multidisciplinary scientific organizations and contributed to the advancement of multiple innovative therapies from early discovery through clinical development and regulatory approval. His extensive background in genetic medicine, RNA technologies, translational science, and platform development is expected to strengthen Stoke’s scientific leadership as the company seeks to broaden its pipeline and accelerate the development of potentially disease-modifying treatments.
Strengthening Scientific Leadership During a Critical Growth Phase
The appointment reflects Stoke Therapeutics’ broader strategy of building a leadership team capable of transforming breakthrough scientific discoveries into medicines that address the root causes of inherited diseases. Unlike conventional treatments that primarily manage symptoms, Stoke’s proprietary approach is designed to restore normal protein production by leveraging RNA medicine to increase expression from healthy copies of genes.
This strategy has already demonstrated encouraging clinical potential through zorevunersen, which is being developed as a first-in-class therapy for Dravet syndrome, a rare and severe genetic epilepsy.
As Chief Scientific Officer, Dr. McCauley will oversee the company’s overall scientific direction, research strategy, platform innovation, and pipeline expansion. His responsibilities will include guiding early discovery programs, advancing translational research, identifying new therapeutic opportunities, and ensuring that Stoke’s RNA medicine platform continues to generate promising clinical candidates across multiple genetic disorders.
Extensive Experience Across Drug Development
Dr. McCauley joins Stoke after building an accomplished career spanning biotechnology startups, established pharmaceutical companies, and emerging platform technology organizations.
Over the course of more than two decades, he has led research teams focused on developing therapies across numerous scientific modalities, including:
- RNA therapeutics
- Genetic medicines
- mRNA technologies
- Rare disease treatments
- Platform biology
- Translational medicine
His experience covers virtually every stage of drug development, from basic scientific discovery through preclinical research, clinical development, regulatory strategy, and commercialization.
This broad expertise positions him to help Stoke continue translating innovative RNA biology into therapies capable of addressing diseases caused by insufficient protein production.
Supporting Expansion Beyond Dravet Syndrome
While Stoke’s most advanced program remains zorevunersen for Dravet syndrome, the company has consistently emphasized that its RNA platform has the potential to treat numerous severe genetic disorders.
Chief Executive Officer Ian F. Smith highlighted the significance of the appointment, noting that Stoke has already established important proof of concept for its technology platform through the clinical progress achieved with zorevunersen.
According to Smith, the investigational medicine has demonstrated the potential to alter the course of Dravet syndrome by addressing the underlying genetic defect rather than simply managing seizures.
He explained that as the company expands beyond its lead program, Dr. McCauley’s deep expertise in translational science and technology development will play an essential role in selecting the strongest scientific opportunities and transforming promising laboratory discoveries into future medicines for patients with severe genetic diseases.
Smith emphasized that McCauley’s scientific leadership will help maximize the full potential of Stoke’s proprietary RNA medicine platform while supporting continued innovation across its expanding research portfolio.
A Unique RNA Medicine Platform
Stoke Therapeutics is pioneering an approach known as Targeted Augmentation of Nuclear Gene Output (TANGO), a proprietary RNA-based technology designed to selectively increase protein production from the healthy copy of a gene.
Many inherited diseases result from mutations that leave patients with only one functioning copy of an essential gene. Although the healthy copy remains present, it often cannot produce enough protein to maintain normal biological function.
Rather than replacing genes or editing DNA, Stoke’s technology aims to increase expression from the existing healthy gene copy, restoring protein levels toward normal.
This approach offers several potential advantages, including:
- Addressing the underlying genetic cause of disease
- Utilizing the body’s natural cellular machinery
- Potential applicability across numerous genetic disorders
- Avoiding permanent modification of DNA
The platform represents one of several emerging RNA medicine technologies seeking to expand treatment options for patients with rare inherited diseases.
Building on Success in Dravet Syndrome
The company’s lead investigational therapy, zorevunersen, has become one of the most closely watched RNA medicine programs for neurological disorders.
Dravet syndrome is a rare developmental and epileptic encephalopathy that typically begins during infancy. The disorder is most commonly caused by mutations in the SCN1A gene, resulting in insufficient production of the sodium channel protein required for normal brain function.
Patients often experience:
- Frequent and prolonged seizures
- Developmental delays
- Cognitive impairment
- Motor dysfunction
- Increased risk of sudden unexpected death in epilepsy (SUDEP)
Current therapies primarily focus on reducing seizure frequency but do not directly address the underlying genetic abnormality.
Zorevunersen is designed to increase production of the SCN1A protein, potentially modifying disease progression rather than simply controlling symptoms.
Its advancement into late-stage development has provided important validation for Stoke’s broader RNA medicine platform.
McCauley Sees Significant Opportunity
Commenting on his appointment, Dr. McCauley said Stoke’s scientific approach immediately stood out because of its potential to fundamentally change treatment strategies for inherited genetic diseases.
Throughout his career, he has worked across numerous therapeutic platforms and drug modalities, giving him a broad perspective on emerging technologies.
He noted that the concept of restoring protein expression through RNA medicine represents an especially promising approach because it directly addresses the biological mechanisms responsible for many severe genetic disorders.
Dr. McCauley also expressed enthusiasm about joining a company that has already advanced a potential first-in-class medicine from early laboratory research into late-stage clinical development.
He said he looks forward to collaborating closely with Stoke’s scientific leadership, including co-founder and Chief Technology Officer Isabel Aznarez, as well as research teams throughout the organization to expand the platform’s capabilities and accelerate development of future therapies.
Leadership Experience Across Biotechnology
Before joining Stoke Therapeutics, Dr. McCauley served as President and Chief Executive Officer of Neptune Bio, where he led strategic planning and advanced therapeutic applications based on the company’s scientific platform.
Prior to Neptune Bio, he held Chief Scientific Officer positions at several biotechnology companies focused on innovative therapeutic technologies.
These include:
- Omega Therapeutics
- Macrolide Pharmaceuticals
- Translate Bio
In each organization, he played a central role in strengthening scientific platforms, expanding research capabilities, and advancing novel therapeutic candidates toward clinical development.
Earlier in his career, Dr. McCauley also held scientific and leadership positions at:
- Shire
- Inotek Pharmaceuticals
- Archemix Corporation
These roles provided extensive experience across rare disease drug development, RNA biology, platform technologies, and translational medicine.
Experience in Rare Genetic Medicines
Among Dr. McCauley’s most significant industry contributions was his six-year tenure at Shire, where he participated in the late-stage development of multiple therapies for rare genetic diseases.
Several of these programs ultimately achieved global regulatory approvals, providing treatments for patients with serious inherited disorders that previously had few therapeutic options.
This experience navigating complex clinical development programs and regulatory pathways is expected to benefit Stoke as it advances zorevunersen through late-stage development and prepares future pipeline candidates for clinical evaluation.
Continuing Scientific Engagement
Beyond his executive leadership roles, Dr. McCauley remains actively involved in the broader biotechnology innovation ecosystem.
He currently serves as a scientific advisor to multiple early-stage biotechnology companies focused on RNA therapeutics and genetic medicine.
These advisory roles allow him to remain closely connected to emerging technologies and evolving scientific approaches that may shape the future of precision medicine.
Strong Academic Foundation
Dr. McCauley’s scientific background is rooted in engineering, physics, and applied research.
He earned both his undergraduate and graduate degrees in Applied and Engineering Physics from Cornell University before completing a Ph.D. in Physics at the University of Alabama at Birmingham.
His interdisciplinary training has supported a career that bridges fundamental science with practical therapeutic development, enabling him to translate complex biological discoveries into clinically relevant innovations.
Positioning Stoke for Future Growth
The appointment of Dr. McCauley signals Stoke Therapeutics’ continued investment in scientific excellence as it evolves from a single-program biotechnology company into a broader developer of RNA medicines targeting multiple severe genetic diseases.
With zorevunersen progressing through late-stage development for Dravet syndrome and additional research programs emerging from its proprietary platform, the company is entering an important period of expansion. By combining experienced scientific leadership with its RNA-based technology, Stoke aims to accelerate the discovery and development of disease-modifying therapies that restore normal protein expression and address the underlying causes of rare inherited disorders. As the field of RNA medicine continues to mature, the company believes its platform and strengthened leadership team position it to contribute meaningfully to the next generation of precision genetic therapies.
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% of patients with Dravet syndrome do not achieve ≥50% 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% 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, and China’s Center for Drug Evaluation has granted zorevunersen Breakthrough Therapy Designation.
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. Zorevunersen is currently in clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.
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.