Encoded Therapeutics to Present ETX101 Clinical Data in Dravet Syndrome at the 2026 ASGCT Presidential Symposium
Encoded Therapeutics Inc. has announced its participation in the American Society of Gene & Cell Therapy 2026 Annual Meeting, where it will present new data highlighting the capabilities of its proprietary vector engineering platform across a range of severe neurological disorders. The meeting, scheduled to take place from May 11–15, 2026, in Boston, Massachusetts, is one of the most prominent global forums for advancements in gene and cell therapy, bringing together researchers, clinicians, and industry leaders.
Encoded Therapeutics will contribute three scientific presentations to the conference program, including a high-profile oral presentation in the Presidential Symposium and two poster presentations. Together, these abstracts reflect the company’s broad approach to developing precision genetic medicines, particularly in areas of high unmet medical need within neurology.
The centerpiece of Encoded’s participation will be its oral presentation focused on ETX101, the company’s lead investigational therapy for SCN1A+ Dravet syndrome. This rare and severe form of epilepsy is caused by mutations in the SCN1A gene, which plays a critical role in regulating neuronal activity. Patients with Dravet syndrome typically experience frequent and treatment-resistant seizures beginning in infancy, along with significant developmental delays and cognitive impairment.
ETX101 is designed as a one-time gene-regulation therapy aimed at addressing the underlying genetic cause of the disease. Unlike traditional gene replacement approaches, ETX101 uses a precision strategy to selectively increase the expression of the SCN1A gene in GABAergic inhibitory neurons. These neurons are essential for maintaining balance in brain activity, and their dysfunction is a key driver of seizures in Dravet syndrome. By restoring SCN1A expression specifically in these cells, ETX101 has the potential to not only reduce seizure frequency but also modify the course of the disease.
The oral presentation will feature interim results from the ongoing POLARIS Phase 1/2 clinical trial, which is evaluating the safety and efficacy of ETX101. The therapy is delivered באמצעות an adeno-associated virus serotype 9 (AAV9) vector, a commonly used platform in gene therapy due to its ability to efficiently target neurons. Early data from POLARIS are expected to provide important insights into the therapy’s clinical impact, including its effects on seizure control and neurodevelopmental outcomes.
Company leadership has expressed optimism about the potential of ETX101 based on the data generated so far. Dr. Sal Rico, Chief Medical Officer of Encoded Therapeutics, emphasized that the findings being presented at ASGCT highlight the therapy’s ability to improve seizure control while also supporting cognitive and learning development in affected children. These outcomes are particularly significant given the profound and lifelong challenges associated with Dravet syndrome.
In addition to the ETX101 presentation, Encoded Therapeutics will showcase two poster presentations that further demonstrate the versatility of its vector engineering platform. One of these focuses on NociPro, a novel regulatory element designed to enable precise targeting of nociceptive neurons—specialized sensory neurons responsible for detecting pain. Chronic pain conditions remain a major clinical challenge, often requiring long-term treatment with medications that can have significant side effects or limited efficacy.
NociPro represents a modality-agnostic promoter platform, meaning it can be applied across different types of gene therapy approaches to achieve targeted gene expression in specific cell types. By enabling selective targeting of pain-sensing neurons, this technology has the potential to support the development of more effective and safer treatments for chronic pain. The preclinical data being presented will highlight how NociPro can be used to achieve precise cellular targeting, a key requirement for advancing gene therapies in this area.
The second poster presentation will focus on an experimental approach for the treatment of Angelman syndrome, another rare neurological disorder characterized by severe developmental delays, lack of speech, seizures, and motor impairment. Angelman syndrome is caused by the loss of function of the UBE3A gene in the brain, typically due to genetic mutations or imprinting defects.
Encoded’s approach involves an intravenously delivered AAV-based vector carrying microRNA (miRNA) designed to “unsilence” the paternal copy of the UBE3A gene. In individuals with Angelman syndrome, the maternal copy of UBE3A is inactive or missing, while the paternal copy is naturally silenced in neurons. By reactivating the paternal gene, this strategy aims to restore functional UBE3A expression and address the root cause of the disorder.
Preclinical data presented in the poster will demonstrate broad neuronal transduction achieved through systemic delivery, as well as successful unsilencing of UBE3A. These findings suggest that the approach could offer a viable path toward a disease-modifying therapy for Angelman syndrome, potentially improving neurological function and quality of life for affected individuals.
Collectively, these presentations underscore Encoded Therapeutics’ commitment to advancing precision genetic medicines באמצעות its proprietary vector engineering technologies. The company’s platform is designed to enable targeted gene regulation, allowing therapies to be tailored to specific cell types and disease mechanisms. This level of precision is increasingly recognized as essential for maximizing efficacy while minimizing off-target effects in gene therapy.
The inclusion of an oral presentation in the Presidential Symposium further highlights the significance of Encoded’s work within the broader gene therapy community. This session typically features some of the most promising and impactful research presented at the conference, providing a platform for sharing breakthroughs that could shape the future of the field.
As gene and cell therapies continue to evolve, the ability to engineer vectors that deliver precise and durable therapeutic effects will be critical. Encoded Therapeutics’ work in areas such as epilepsy, chronic pain, and neurodevelopmental disorders reflects a growing focus on addressing complex neurological conditions באמצעות innovative genetic approaches.
In summary, Encoded’s participation at ASGCT 2026 will showcase the breadth and depth of its pipeline, from clinical-stage programs like ETX101 to preclinical innovations such as NociPro and miRNA-based therapies for Angelman syndrome. These efforts highlight the company’s broader vision of delivering one-time, precision genetic treatments that can transform outcomes for patients with severe neurological diseases.
About Encoded Therapeutics
Encoded Therapeutics is a clinical-stage biotechnology company developing one-time precision genetic medicines for severe monogenic and common neurological disorders. The company’s vector engineering platform enables highly targeted and cell-type-selective control of gene expression in the brain and peripheral nervous system, allowing potent and precise modulation of disease-relevant genes to address underlying disease biology. Encoded’s end‑to‑end innovation engine—spanning discovery, development, and in‑house GMP manufacturing—creates a streamlined path to advance a diversified pipeline of one‑time treatments across a broad range of neurological conditions. Encoded is driven by a mission to meaningfully improve the lives of patients and families affected by devastating neurological disorders.
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