{"id":8898,"date":"2026-10-30T14:28:56","date_gmt":"2026-10-30T11:28:56","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8898"},"modified":"2026-10-30T14:35:21","modified_gmt":"2026-10-30T11:35:21","slug":"pepr-124-rt001-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/es\/pepr-124-rt001-duchenne\/","title":{"rendered":"PEPR-124 (RT001): Un f\u00e1rmaco candidato para la distrofia muscular de Duchenne, descubierto mediante IA y que no depende de mutaciones espec\u00edficas."},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>PEPR-124 (also designated as RT001) is an AI-discovered drug candidate developed by Peptris Technologies, a Bengaluru startup based in India, specifically targeting Duchenne Muscular Dystrophy (DMD).<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Founded in 2019 and operating from C-CAMP, Peptris uses artificial intelligence and generative chemistry to design optimized molecules for once &#8220;undruggable&#8221; diseases like rare illnesses, cancer, and inflammation. Supported by a fresh \u20b986 crore grant (approximately 9,047,200 USD) from the RDI Fund to expand into a full-scale drug discovery company while leveraging India&#8217;s low-cost ecosystem, Peptris has successfully licensed this repurposed asset globally. As Co-founder and CEO Venkatasubramanian Narayanan (Venkat) highlights, &#8220;AI speeds things up, but science still leads the way.&#8221; This advancement marks a vital breakthrough for the estimated 400,000 DMD patients in India who previously faced highly limited therapeutic options.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Tabla de contenido<\/h2><nav><ul><li><a href=\"#discovery-and-structural-overview\">PEPR-124: Discovery and Structural Overview<\/a><\/li><li><a href=\"#mechanism-of-action-mutation-agnostic-utrophin-upregulation\">Mechanism of Action: Mutation-Agnostic Utrophin Upregulation<\/a><\/li><li><a href=\"#preclinical-efficacy-and-safety-data\">Preclinical Efficacy and Safety Data<\/a><\/li><li><a href=\"#commercialization-strategic-licensing-and-regulatory-status\">Commercialization, Strategic Licensing, and Regulatory Status<\/a><\/li><li><a href=\"#future-outlook-the-hybrid-clinical-path\">Future Outlook: The Hybrid Clinical Path<\/a><\/li><li><a href=\"#references\">References<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"discovery-and-structural-overview\" class=\"wp-block-heading\">PEPR-124: Discovery and Structural Overview<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>PEPR-124 was originally discovered by Peptris Technologies, deploying its proprietary computational platform (pepAI) at C-CAMP to map extensive chemical spaces and isolate functional small molecules.<\/strong> Driven by the recent \u20b986 crore RDI Fund grant, the company is actively expanding into a full-scale drug discovery firm while leveraging the lower operational costs in India to build lasting, accessible solutions for hard-to-treat illnesses.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Unlike novel synthesized compounds that carry high translational risks, PEPR-124 is a repurposed asset. This strategic designation means the foundational molecule possesses established baseline safety profiles, drastically shortening the typical decade-long preclinical validation timeline and mitigating toxicity or instability failures during development.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"mechanism-of-action-mutation-agnostic-utrophin-upregulation\" class=\"wp-block-heading\">Mechanism of Action: Mutation-Agnostic Utrophin Upregulation<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMD is a catastrophic, progressive neuromuscular disorder caused by genetic errors that prevent the production of functional dystrophin, a critical protein stabilizing muscle cell membranes. Standard gene correction techniques (such as exon-skipping) are limited because they target specific genetic variants.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>PEPR-124 bypasses this constraint by implementing a mutation-agnostic therapeutic approach:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Target Protein:<\/strong> It focuses on upregulating utrophin, a structural analogue protein functionally identical to dystrophin in embryonic development.<\/li>\n\n\n\n<li><strong>Cellular Compensation:<\/strong> By triggering a permanent increase in local utrophin levels, it compensates for the missing dystrophin framework, safeguarding muscle fibers against mechanical strain regardless of the child&#8217;s exact genetic deletion sequence.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"preclinical-efficacy-and-safety-data\" class=\"wp-block-heading\">Preclinical Efficacy and Safety Data<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Preclinical trials presented at major scientific symposiums, including the Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference, demonstrate significant therapeutic markers:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>In Vitro Assays:<\/strong> PEPR-124 successfully achieved dose-dependent utrophin upregulation in C2C12 myoblasts as well as human induced pluripotent stem cells (hIPSCs) derived directly from human DMD patients.<\/li>\n\n\n\n<li><strong>In Vivo Muscle Function:<\/strong> In severe D2-Mdx mouse models evaluated over 28-day and 90-day intervals (dosed at 25\u2013100 mg\/kg), the compound generated major improvements in global muscle strength, grip strength, hanging latency tests, and general treadmill performance.<\/li>\n\n\n\n<li><strong>Combination Synergies:<\/strong> When co-administered with standard-of-care corticosteroid deflazacort, PEPR-124 yielded superior structural muscle protection, improved hanging endurance, and noticeably decreased serum creatine kinase (a gold-standard blood biomarker for ongoing muscle destruction).<\/li>\n\n\n\n<li><strong>Perfil de seguridad:<\/strong> Crucially, PEPR-124 showed no adverse events regarding body weight growth or metabolic toxicity, presenting a favorable safety margin compared to long-term corticosteroid monotherapy.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"commercialization-strategic-licensing-and-regulatory-status\" class=\"wp-block-heading\">Commercialization, Strategic Licensing, and Regulatory Status<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">To accelerate international clinical development, Peptris Technologies, signed a global licensing agreement with Revio Therapeutics, a U.S.-based specialty pharmaceutical startup specializing in optimizing approved medicines.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Market Allocations:<\/strong> Revio Therapeutics holds exclusive development and commercialization rights globally, while Peptris retains commercial rights specifically for the BRICS markets.<\/li>\n\n\n\n<li><strong>FDA Orphan Drug Designation:<\/strong> The asset achieved a vital regulatory milestone when the U.S. FDA granted Orphan Drug Designation (ODD) to RT001\/PEPR-124. This regulatory status provides development incentives, tax credits for clinical testing, waiver of PDUFA fees, and potential post-approval market exclusivity.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"future-outlook-the-hybrid-clinical-path\" class=\"wp-block-heading\">Future Outlook: The Hybrid Clinical Path<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>As of late 2026, PEPR-124 is classified as a Phase 2-ready asset. Revio Therapeutics is leveraging the FDA&#8217;s regulatory 505(b)(2) hybrid pathway.<\/strong> This allows the sponsor to reference existing safety data for the foundational small molecule, eliminating the requirement for redundant Phase 1 safety trials in healthy volunteers. Revio is preparing pre-IND (Investigational New Drug) meetings with the U.S. FDA to finalize the architecture of the upcoming Phase 2 clinical trials in children diagnosed with DMD.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"references\" class=\"wp-block-heading\">References<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Revio Therapeutics. <em>&#8220;Revio Partners with Peptris to License RT001 (PEPR124) for Muscular Dystrophy.&#8221;<\/em> <a href=\"https:\/\/www.reviotx.com\/blog\/revio-partners-with-peptris\" target=\"_blank\" rel=\"noopener\">Revio Therapeutics Blog<\/a>.<\/li>\n\n\n\n<li>YourStory. <em>&#8220;Peptris uses AI and licenses Duchenne muscular dystrophy candidate; wins \u20b986cr RDI grant.&#8221;<\/em> <a href=\"https:\/\/yourstory.com\/2026\/08\/peptris-ai-drug-discovery\" target=\"_blank\" rel=\"noopener\">YourStory Tech Media<\/a>.<\/li>\n\n\n\n<li>Clival News. <em>&#8220;Peptris Licenses India&#8217;s First AI-Discovered Drug to Revio Therapeutics for Duchenne Muscular Dystrophy.&#8221;<\/em> <a href=\"https:\/\/clival.com\/news\/peptris-licenses-indias-first-ai-discovered-drug-to-revio-therapeutics\" target=\"_blank\" rel=\"noopener\">Clival Biotech Markets<\/a>.<\/li>\n\n\n\n<li>Revio Therapeutics. <em>&#8220;RT001 Regulatory Milestone: FDA Grants Orphan Drug Designation for Duchenne Muscular Dystrophy Program.&#8221;<\/em> <a href=\"https:\/\/www.reviotx.com\/blog\/%20rt001-regulatory-milestone\" target=\"_blank\" rel=\"noopener\">Revio Therapeutics News<\/a>.<\/li>\n\n\n\n<li>Muscular Dystrophy Association (MDA). <em>&#8220;PEPR124 (RT001): A repurposed small molecule drug as a mutation agnostic and safe therapeutic for Duchenne Muscular Dystrophy.&#8221;<\/em> <a href=\"https:\/\/www.mdaconference.org\/abstract-library\/pepr124-rt001-a-repurposed-small-molecule-drug-as-a-mutation-agnostic-and-safe-therapeutic-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener\">MDA Conference Abstract Library<\/a>.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>PEPR-124 (also designated as RT001) is an AI-discovered drug candidate developed by Peptris Technologies, a Bengaluru startup based in India, specifically targeting Duchenne Muscular Dystrophy (DMD). Founded in 2019 and operating from C-CAMP, Peptris uses artificial intelligence and generative chemistry to design optimized molecules for once &#8220;undruggable&#8221; diseases like rare illnesses, cancer, and inflammation. Supported [&hellip;]<\/p>\n","protected":false},"author":3,"featured_media":8901,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[83,937,938,939,940,925],"class_list":["post-8898","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-india","tag-pepr-124","tag-peptris-technologies","tag-revio-therapeutics","tag-rt001","tag-utrophin"],"subtitle":"Could an AI-discovered drug offer a new approach to Duchenne muscular dystrophy? PEPR-124 (RT001), developed by Peptris Technologies, aims to boost utrophin regardless of the underlying mutation. Discover its preclinical findings and development prospects.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/8898","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/comments?post=8898"}],"version-history":[{"count":4,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/8898\/revisions"}],"predecessor-version":[{"id":8905,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/8898\/revisions\/8905"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media\/8901"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media?parent=8898"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/categories?post=8898"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/tags?post=8898"}],"curies":[{"name":"gracias","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}