{"id":4301,"date":"2026-05-12T15:00:00","date_gmt":"2026-05-12T13:00:00","guid":{"rendered":"http:\/\/stocks-future.com\/?guid=95dbfe0ed5b80864637eba75ae92d78c"},"modified":"2026-05-12T15:00:00","modified_gmt":"2026-05-12T13:00:00","slug":"encoded-therapeutics-presents-new-preclinical-data-at-asgct-2026-highlighting-precision-vector-engineering-across-neurology-programs","status":"publish","type":"post","link":"https:\/\/stocks-future.com\/?p=4301","title":{"rendered":"Encoded Therapeutics Presents New Preclinical Data at ASGCT 2026 Highlighting Precision Vector Engineering Across Neurology Programs"},"content":{"rendered":"

\n\u2013 NociPro enables highly selective nociceptor targeting and potent NaV<\/sub>1.7 knockdown in rodents and non-human primates, supporting development candidate ETX301 for post\u2011amputation neuroma pain \u2013<\/i><\/p>

\n\u2013 Intravenous AAV-miRNA approach for Angelman syndrome is well-tolerated and achieves broad unsilencing of UBE3A in non\u2011human primates, with approximately 57% increased UBE3A expression across multiple disease-relevant brain regions \u2013<\/i><\/p>

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--#ASGCT2026<\/a>--Encoded Therapeutics Inc. (\u201cEncoded\u201d), 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\u2019s vector engineering platform, which is designed to achieve cell-type-selective regulation of disease\u2011relevant genes across neurological disorders and to enable differentiated, one\u2011time genetic medicines. Posters will be available 5:00-6:30 p.m. ET in the Poster Hall.<\/p><\/a><\/a>

\n\u201cThe data we\u2019re 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,\u201d said Stephanie Tagliatela, Co-founder and Chief Scientific Officer of Encoded. \u201cFrom 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\u2011time 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.\u201d<\/p>

\nNociPro: A Novel Modality\u2011Agnostic Promoter Platform for Precise Cellular Targeting of Nociceptor Sensory Neurons in Gene Therapy for Chronic Pain (Poster #1312):<\/b><\/p>

\nThe poster highlights NociPro\u2014a regulatory element derived from human genomic sequences that enables potent and selective targeting of pain\u2011sensing nociceptive neurons. Developed using Encoded\u2019s precision vector engineering platform, NociPro is a core component of ETX301, the company\u2019s vectorized miRNA candidate targeting SCN9A <\/i>(miRNAhSCN9A<\/sup>) to treat post\u2011amputation neuroma pain.<\/p>

\nIn new preclinical studies across mouse, human iPSC\u2011derived 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\u2011neuronal benchmark promoters. Across species, NociPro demonstrated highly selective DRG\u2011restricted activity, with no detectable expression in the CNS. When coupled with miRNAhSCN9A<\/sup>, NociPro achieved NaV<\/sub>1.7 protein reduction comparable to a ubiquitous benchmark promoter in NHPs, while demonstrating improved tolerability.<\/p>

\nBuilding on prior work that demonstrated robust, gene\u2011selective knockdown of SCN9A<\/i> (NaV<\/sub>1.7) 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.<\/p>

\nNeuronal Transduction and UBE3A<\/i> Unsilencing for the Treatment of Angelman Syndrome (Poster #1468):<\/b><\/p>

\nThe poster features preclinical proof\u2011of\u2011concept data for a one\u2011time, intravenously delivered vectorized miRNA gene therapy designed to unsilence the UBE3A<\/i> gene. By pairing an optimized miRNA candidate with an externally-developed blood\u2011brain\u2011barrier\u2013crossing AAV variant, the approach achieved widespread miRNA expression across disease\u2011relevant brain regions in NHPs.<\/p>

\nCorresponding target engagement included a 57% increase in UBE3A<\/i> expression and a 24% increase in UBE3A<\/i> protein levels across disease-relevant brain regions, including cortex, hippocampus, and thalamus. This therapy was well tolerated in a 60\u2011day study with no adverse findings. These results highlight the potential for a non\u2011invasive, durable therapeutic approach that addresses the underlying cause of Angelman syndrome.<\/p>

\nAbout Encoded Therapeutics<\/strong><\/p>

\nEncoded Therapeutics is a clinical-stage biotechnology company developing one-time precision genetic medicines for severe monogenic and common neurological disorders. The company\u2019s 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\u2019s end\u2011to\u2011end innovation engine\u2014spanning discovery, development, and in\u2011house GMP manufacturing\u2014creates a streamlined path to advance a diversified pipeline of one\u2011time 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<\/b><\/a>.<\/p> Contacts<\/b>

\nInvestors\/Business Development\nJennifer Gorzelany\ncommunications@encoded.com<\/a><\/p>

\nMedia\nLori Rosen\nlori@redhousecomms.com<\/a><\/p>","protected":false},"excerpt":{"rendered":"

\u2013 NociPro enables highly selective nociceptor targeting and potent NaV1.7 knockdown in rodents and non-human primates, supporting development candidate ETX301 for post\u2011amputation neuroma pain \u2013
\n\u2013 Intravenous AAV-miRNA approach for Angelman syndrome is…<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-4301","post","type-post","status-publish","format-standard","hentry","category-infos-businesswire"],"_links":{"self":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts\/4301","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=4301"}],"version-history":[{"count":1,"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts\/4301\/revisions"}],"predecessor-version":[{"id":4302,"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts\/4301\/revisions\/4302"}],"wp:attachment":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=4301"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=4301"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=4301"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}