Encoded Therapeutics Inc. (“Encoded”), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today will present new preclinical data across two posters at the 29th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT). The presentations expand the growing body of evidence supporting the company’s vector engineering platform, which is designed to achieve cell-type-selective regulation of disease‑relevant genes across neurological disorders and to enable differentiated, one‑time genetic medicines. Posters will be available 5:00-6:30 p.m. ET in the Poster Hall.
“The data we’re presenting at ASGCT further demonstrate how our vector engineering platform can be tuned to achieve the level of cellular precision required to address the diverse biological challenges of neurological disorders,” said Stephanie Tagliatela, Co-founder and Chief Scientific Officer of Encoded. “From highly selective nociceptor targeting in the peripheral nervous system to broad neuronal transduction and UBE3A unsilencing in the CNS, these findings reinforce the potential for one‑time genetic medicines that directly address the underlying mechanisms of disease. Our goal is to build therapies that are both biologically precise and clinically meaningful, and these new data move us closer to that vision.”
NociPro: A Novel Modality‑Agnostic Promoter Platform for Precise Cellular Targeting of Nociceptor Sensory Neurons in Gene Therapy for Chronic Pain (Poster #1312):
The poster highlights NociPro—a regulatory element derived from human genomic sequences that enables potent and selective targeting of pain‑sensing nociceptive neurons. Developed using Encoded’s precision vector engineering platform, NociPro is a core component of ETX301, the company’s vectorized miRNA candidate targeting SCN9A (miRNAhSCN9A) to treat post‑amputation neuroma pain.
In new preclinical studies across mouse, human iPSC‑derived neurons, and non-human primates (NHPs), NociPro drove robust and selective transgene expression in pain-sensing dorsal root ganglion (DRG) nociceptors, with activity comparable to ubiquitous and pan‑neuronal benchmark promoters. Across species, NociPro demonstrated highly selective DRG‑restricted activity, with no detectable expression in the CNS. When coupled with miRNAhSCN9A, NociPro achieved NaV1.7 protein reduction comparable to a ubiquitous benchmark promoter in NHPs, while demonstrating improved tolerability.
Building on prior work that demonstrated robust, gene‑selective knockdown of SCN9A (NaV1.7) using an engineered vectorized miRNA, these findings underscore the potential of NociPro to enhance the precision and therapeutic index of gene therapies for chronic pain.
Neuronal Transduction and UBE3A Unsilencing for the Treatment of Angelman Syndrome (Poster #1468):
The poster features preclinical proof‑of‑concept data for a one‑time, intravenously delivered vectorized miRNA gene therapy designed to unsilence the UBE3A gene. By pairing an optimized miRNA candidate with an externally-developed blood‑brain‑barrier–crossing AAV variant, the approach achieved widespread miRNA expression across disease‑relevant brain regions in NHPs.
Corresponding target engagement included a 57% increase in UBE3A expression and a 24% increase in UBE3A protein levels across disease-relevant brain regions, including cortex, hippocampus, and thalamus. This therapy was well tolerated in a 60‑day study with no adverse findings. These results highlight the potential for a non‑invasive, durable therapeutic approach that addresses the underlying cause of Angelman syndrome.
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. For more information, please visit www.encoded.com.
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