{"id":8550,"date":"2026-08-21T12:50:49","date_gmt":"2026-08-21T09:50:49","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8550"},"modified":"2026-08-21T12:50:52","modified_gmt":"2026-08-21T09:50:52","slug":"hlb-panagene-pna-aoc-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ru\/hlb-panagene-pna-aoc-dmd\/","title":{"rendered":"HLB Panagene \u0438\u0441\u043f\u043e\u043b\u044c\u0437\u0443\u0435\u0442 PNA-AOC \u0432 \u043a\u0430\u0447\u0435\u0441\u0442\u0432\u0435 \u043d\u043e\u0432\u043e\u0433\u043e \u043f\u043e\u0434\u0445\u043e\u0434\u0430 \u043a \u043b\u0435\u0447\u0435\u043d\u0438\u044e \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0438 \u0414\u044e\u0448\u0435\u043d\u043d\u0430."},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne muscular dystrophy (DMD) remains a major challenge for drug developers because many therapeutic approaches have struggled to deliver sufficient amounts of a treatment to skeletal and cardiac muscle while maintaining an acceptable safety profile. <strong>Against this backdrop, South Korean biotechnology company HLB Panagene is exploring a different strategy based on a peptide nucleic acid (PNA)-based antibody-oligonucleotide conjugate (AOC) platform.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">AOC is an emerging therapeutic modality derived from the concept of antibody-drug conjugates (ADCs). Instead of carrying a conventional cytotoxic drug, an AOC uses an antibody to deliver a nucleic-acid-based therapeutic payload to specific cells. The goal is to combine the targeting capability of antibodies with the ability of nucleic acids to regulate gene expression.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">One of the major challenges with nucleic-acid therapeutics is delivery. These molecules generally have limited ability to cross cell membranes. Even when they enter cells through antibody-mediated delivery, they can become trapped inside endosomes or be degraded before reaching their intended intracellular target. <strong>HLB Panagene believes that PNA could help address some of these limitations because of its structural stability and strong target-binding characteristics.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>The company has selected DMD as the first disease indication for validating its PNA-AOC platform.<\/strong> Its approach is particularly relevant to exon-skipping therapy, where an oligonucleotide is designed to modify dystrophin pre-mRNA processing and allow cells to produce a shorter but potentially functional dystrophin protein. HLB Panagene is pursuing an AOC architecture intended to improve delivery of an exon-skipping PNA to muscle tissue. <strong>Industry reports indicate that a transferrin receptor 1 (TfR1) antibody is being explored as part of the delivery strategy, with a preclinical proof-of-concept targeted for 2026.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>The strategy comes as several existing DMD therapeutic approaches have encountered limitations involving efficacy, delivery or safety.<\/strong> Other companies are also developing targeted oligonucleotide delivery technologies, including AOC-based programs, suggesting that delivery efficiency has become one of the central battlegrounds in next-generation DMD drug development.<\/p>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><thead><tr><th>\u041f\u043e\u0434\u0445\u043e\u0434<\/th><th>Payload<\/th><th>Delivery strategy<\/th><th>DMD target<\/th><th>Development stage<\/th><\/tr><\/thead><tbody><tr><td>Traditional exon skipping<\/td><td>\u041e\u0423\u041f<\/td><td>Systemic<\/td><td>Specific exons<\/td><td>\u041e\u0434\u043e\u0431\u0440\u0435\u043d\u043d\u044b\u0439<\/td><\/tr><tr><td>AOC approach<\/td><td>Oligonucleotide<\/td><td>Antibody-mediated<\/td><td>Muscle<\/td><td>Clinical development<\/td><\/tr><tr><td>\u041f\u041d\u0410-\u0410\u041e\u041a<\/td><td>PNA<\/td><td>Antibody-mediated<\/td><td>Muscle<\/td><td>Early\/preclinical<\/td><\/tr><tr><td>\u0413\u0435\u043d\u043d\u0430\u044f \u0442\u0435\u0440\u0430\u043f\u0438\u044f<\/td><td>\u041c\u0438\u043a\u0440\u043e\u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u043d<\/td><td>\u0410\u0410\u0412<\/td><td>Broad muscle<\/td><td>Clinical\/approved depending on product<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>However, HLB Panagene&#8217;s PNA-AOC program remains early-stage.<\/strong> There is currently no approved PNA-AOC therapy for DMD, and the company&#8217;s approach still needs to demonstrate effective muscle delivery, exon-skipping activity, dystrophin restoration, safety and ultimately clinical benefit in human studies.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>For DMD families, therefore, PNA-AOC should be viewed not as an available treatment, but as an experimental therapeutic platform worth monitoring.<\/strong> Its potential significance lies in attempting to solve one of the fundamental problems facing nucleic-acid therapies: getting the right therapeutic molecule to the right muscle cells at an effective and sustainable level.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0423\u0437\u043d\u0430\u0439\u0442\u0435 \u0441\u0435\u0439\u0447\u0430\u0441: <a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/\">\u041a\u043b\u0438\u043d\u0438\u0447\u0435\u0441\u043a\u0438\u0435 \u0438\u0441\u043f\u044b\u0442\u0430\u043d\u0438\u044f DMD<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Duchenne muscular dystrophy (DMD) remains a major challenge for drug developers because many therapeutic approaches have struggled to deliver sufficient amounts of a treatment to skeletal and cardiac muscle while maintaining an acceptable safety profile. Against this backdrop, South Korean biotechnology company HLB Panagene is exploring a different strategy based on a peptide nucleic acid [&hellip;]<\/p>\n","protected":false},"author":9,"featured_media":8551,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[872,873,158],"class_list":["post-8550","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-hlb-panagene","tag-pna-aoc","tag-south-korea"],"subtitle":"Duchenne muscular dystrophy (DMD) therapy is entering a new era of targeted delivery. HLB Panagene is exploring PNA-AOC technology to improve treatment precision. Could this approach overcome one of DMD therapy\u2019s biggest challenges\u2014getting the right treatment into muscle cells?","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/posts\/8550","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/users\/9"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/comments?post=8550"}],"version-history":[{"count":1,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/posts\/8550\/revisions"}],"predecessor-version":[{"id":8552,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/posts\/8550\/revisions\/8552"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/media\/8551"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/media?parent=8550"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/categories?post=8550"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/tags?post=8550"}],"curies":[{"name":"WP","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}