SOUTH SAN FRANCISCO, Calif., April 27, 2026

Encoded Therapeutics announced its participation in the 29th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) 2026, where it will present new interim clinical data for ETX101, a precision gene-regulation therapy for Dravet syndrome, alongside additional platform-focused research. The presentations highlight the company’s advancing leadership in gene therapy and vector engineering technologies targeting severe neurological disorders.

ETX101 Demonstrates Promise in Dravet Syndrome

The highlight of the presentation will be an oral session in the Presidential Symposium, featuring interim results from the ongoing POLARIS Phase 1/2 clinical trials evaluating ETX101. This investigational therapy is designed as a one-time gene-regulation treatment that selectively increases SCN1A gene expression in GABAergic inhibitory neurons, addressing the root cause of Dravet syndrome. Early findings suggest that ETX101 has the potential to improve seizure control and support cognitive and developmental outcomes, representing a disease-modifying approach rather than symptomatic management.

Dravet syndrome is a severe, rare genetic epilepsy that begins in infancy and is associated with frequent seizures, developmental delays, and high unmet medical need. Current treatment options are limited and often fail to provide sustained control. By targeting the underlying genetic mechanism, ETX101 could offer a transformational therapeutic strategy, especially as a single-administration therapy designed for long-term benefit. The interim POLARIS data presented at ASGCT are expected to provide further insights into safety, tolerability, and early efficacy signals in affected pediatric patients..

Expanding Platform Capabilities in Neurology

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In addition to the ETX101 clinical update, Encoded Therapeutics will present two scientific posters demonstrating the broader capabilities of its proprietary vector engineering platform. One presentation introduces NociPro, a novel regulatory element designed to precisely target nociceptive neurons, opening new possibilities for gene therapy approaches in chronic pain management. This platform aims to enable cell-type-specific gene expression control, enhancing both safety and therapeutic precision.

Another poster highlights an intravenous AAV-miRNA-based approach for the treatment of Angelman syndrome, a rare neurogenetic disorder. The approach is designed to unsilence the UBE3A gene, restoring its function across neuronal populations. Preclinical data demonstrate broad neuronal transduction and effective gene reactivation, suggesting strong potential for future clinical translation. Together, these studies underscore the versatility of Encoded’s platform in addressing diverse neurological conditions through targeted genetic modulation.

Driving Innovation in One-Time Genetic Medicines

Encoded Therapeutics continues to position itself at the forefront of next-generation genetic medicine development, with a focus on delivering precision, one-time therapies for complex neurological diseases. Its integrated platform combines advanced vector design, targeted gene regulation, and in-house GMP manufacturing, enabling efficient progression from discovery to clinical development.

The company’s participation in ASGCT 2026 reflects its commitment to advancing innovative therapeutic solutions that address the underlying biology of disease. By leveraging its cell-type-specific gene control technologies, Encoded aims to overcome traditional limitations of gene therapy, such as off-target effects and limited durability.

As the field of gene and cell therapy continues to evolve, the data presented at ASGCT highlight the growing potential of precision genetic interventions to transform treatment paradigms. ETX101 and the broader pipeline represent a significant step forward in the development of durable, disease-modifying therapies for patients with rare and severe neurological disorders.

Encoded’s ongoing clinical and preclinical progress reinforces its mission to deliver impactful therapies that improve patient outcomes and quality of life, particularly in areas where current treatment options remain insufficient.

Source: Encoded Therapeutics press release