{"id":31340,"date":"2025-03-31T17:16:00","date_gmt":"2025-03-31T11:46:00","guid":{"rendered":"https:\/\/www.delveinsight.com\/blog\/?p=31340"},"modified":"2025-03-20T16:16:57","modified_gmt":"2025-03-20T10:46:57","slug":"mps-i-treatment-pipeline","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/mps-i-treatment-pipeline","title":{"rendered":"Mucopolysaccharidosis Type I Treatment Pipeline \u2014 4 Promising Therapies on the Way"},"content":{"rendered":"<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_76 counter-hierarchy ez-toc-counter ez-toc-white ez-toc-container-direction\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Table of Contents<\/p>\n<label for=\"ez-toc-cssicon-toggle-item-69f2fe229cf53\" class=\"ez-toc-cssicon-toggle-label\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/label><input type=\"checkbox\"  id=\"ez-toc-cssicon-toggle-item-69f2fe229cf53\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.delveinsight.com\/blog\/mps-i-treatment-pipeline\/#4_Promising_MPS_I_Therapies_in_Development\" >4 Promising MPS I Therapies in Development<\/a><ul class='ez-toc-list-level-3' ><li class='ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.delveinsight.com\/blog\/mps-i-treatment-pipeline\/#Orchard_TherapeuticsKyowa_Kirins_OTL-203\" >Orchard Therapeutics\/Kyowa Kirin\u2019s OTL-203<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.delveinsight.com\/blog\/mps-i-treatment-pipeline\/#JCR_Pharmaceuticals_JR-171\" >JCR Pharmaceuticals\u2019 JR-171<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/www.delveinsight.com\/blog\/mps-i-treatment-pipeline\/#REGENXBIONippon_Shinyakus_RGX-111\" >REGENXBIO\/Nippon Shinyaku\u2019s RGX-111<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/www.delveinsight.com\/blog\/mps-i-treatment-pipeline\/#Immusofts_ISP-001\" >Immusoft\u2019s ISP-001<\/a><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/www.delveinsight.com\/blog\/mps-i-treatment-pipeline\/#What_Lies_Ahead_in_MPS_I_Treatment\" >What Lies Ahead in MPS I Treatment?<\/a><\/li><\/ul><\/nav><\/div>\n\n<p>MPS I treatment typically involves Hematopoietic Stem Cell Transplantation (HSCT), Enzyme Replacement Therapy (ERT), or a combination of both, along with supportive care such as pain management, anti-inflammatory medications, oxygen therapy, and surgery. While these <a href=\"https:\/\/www.delveinsight.com\/blog\/mucopolysaccharidosis-types-and-treatment-strategies\">MPS I treatments<\/a> help manage symptoms and improve survival, they cannot reverse existing damage. Early intervention is essential to achieve the best possible outcomes.<\/p>\n\n\n\n<p><strong>ALDURAZYME (developed by BioMarin and Sanofi)<\/strong> is the only therapy approved by the US FDA for treating MPS I. ALDURAZYME was approved in the US and EU4 (France, Germany, Italy, and Spain) and the UK in 2003 and in Japan in 2006. It is indicated for patients with the Hurler and Hurler-Scheie forms of MPS I, as well as those with the Scheie form who have moderate to severe symptoms. The treatment includes a boxed warning about potential risks.<\/p>\n\n\n\n<p>Although its patent has expired, no biosimilars are currently available, making ALDURAZYME the only enzyme replacement therapy for MPS I on the market. However, several companies are working with their lead assets that will give tough competition to BioMarin\/Sanofi\u2019s ALDURAZYME.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-4-promising-mps-i-therapies-in-development\"><span class=\"ez-toc-section\" id=\"4_Promising_MPS_I_Therapies_in_Development\"><\/span><strong>4 Promising MPS I Therapies in Development<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>The MPS I treatment pipeline remains limited, with only a few new therapies currently in development. Leading companies driving progress include <strong>Kyowa Kirin and Orchard Therapeutics<\/strong> (developing OTL-203, a gene therapy targeting the underlying enzyme deficiency), <strong>JCR Pharmaceuticals<\/strong> (advancing JR-171, an enzyme replacement therapy that can cross the blood-brain barrier), <strong>Immusoft <\/strong>(developing ISP-001, a cell-based platform for continuous enzyme production), and <strong>REGENXBIO <\/strong>(working on RGX-111, a gene therapy designed to deliver a functional copy of the IDUA gene).&nbsp;<\/p>\n\n\n\n<p><em>These innovative treatments aim to improve neurological function and overall patient outcomes, addressing critical unmet needs in the MPS I community. Let\u2019s get into detail about these 4 MPS I therapies in development.<\/em><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"h-orchard-therapeutics-kyowa-kirin-s-otl-203\"><span class=\"ez-toc-section\" id=\"Orchard_TherapeuticsKyowa_Kirins_OTL-203\"><\/span><strong>Orchard Therapeutics\/Kyowa Kirin\u2019s OTL-203<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>OTL-203<\/strong> is a one-time <strong>gene therapy<\/strong> for MPS IH that uses a patient\u2019s own hematopoietic stem and progenitor cells (HSPCs). These cells are collected from mobilized peripheral blood and genetically modified ex vivo with a lentiviral vector carrying functional IDUA complementary DNA to restore enzyme production and reduce GAG accumulation. Developed in collaboration with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), OTL-203 is currently in <strong>Phase III <\/strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/mucopolysaccharidosis-i-pipeline-insight\"><strong>MPS I clinical trials<\/strong><\/a> in North America and Europe.<\/p>\n\n\n\n<p>Orchard Therapeutics plans to submit a US application in 2028, with potential approval expected in 2029 under priority review. OTL-203 has received <strong>Fast Track Designation (FTD), Orphan Drug Designation (ODD), and Rare Pediatric Disease Designation (RPDD)<\/strong> from the FDA, as well as <strong>PRIME status<\/strong> from the EMA.<\/p>\n\n\n\n<p>At the <strong>21st Annual WORLDSymposium <\/strong>in February 2025, Orchard Therapeutics presented updated findings from a proof-of-concept study on OTL-203, highlighting improvements in neurological, skeletal, and other clinical outcomes. Additionally, in February 2024, the company announced the first patient\u2019s randomization in the registrational trial of OTL-203 for MPS IH, marking a key milestone in its clinical development.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"h-jcr-pharmaceuticals-jr-171\"><span class=\"ez-toc-section\" id=\"JCR_Pharmaceuticals_JR-171\"><\/span><strong>JCR Pharmaceuticals\u2019 JR-171<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>JR-171 (lepunafusp alfa)<\/strong> is an advanced <strong>enzyme replacement therapy (ERT)<\/strong> designed to address central nervous system (CNS) complications in MPS I. It is a recombinant fusion protein that combines an antibody targeting the human transferrin receptor with IDUA, the deficient enzyme in MPS I patients. By leveraging transferrin receptor-mediated transcytosis, JR-171 effectively crosses the blood-brain barrier (BBB), addressing a critical gap in CNS treatment.<\/p>\n\n\n\n<p>Developed using JCR Pharmaceuticals\u2019 proprietary <strong>J-Brain Cargo and J-MIG System platforms<\/strong>, JR-171 has completed a 13-week <strong>Phase I\/II clinical trial<\/strong> in Japan and the US, with an extension study currently underway. JR-171 is being developed through licensing partnerships, with ongoing collaboration discussions. It has received <strong>Fast Track Designation (FTD) and Orphan Drug Designation (ODD)<\/strong> from both the FDA and EMA.<\/p>\n\n\n\n<p>In September 2024, JCR Pharmaceuticals presented data at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, highlighting improvements in both neurobehavioral and somatic symptoms in MPS I patients treated with JR-171.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img decoding=\"async\" width=\"1024\" height=\"357\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155456\/Emerging-Mucopolysaccharidosis-Type-I-Therapies-1024x357.png\" alt=\"Emerging-Mucopolysaccharidosis-Type-I-Therapies\" class=\"wp-image-31335\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155456\/Emerging-Mucopolysaccharidosis-Type-I-Therapies-1024x357.png 1024w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155456\/Emerging-Mucopolysaccharidosis-Type-I-Therapies-300x105.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155456\/Emerging-Mucopolysaccharidosis-Type-I-Therapies-150x52.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155456\/Emerging-Mucopolysaccharidosis-Type-I-Therapies-768x268.png 768w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155456\/Emerging-Mucopolysaccharidosis-Type-I-Therapies-1536x536.png 1536w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155456\/Emerging-Mucopolysaccharidosis-Type-I-Therapies-2048x714.png 2048w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"h-regenxbio-nippon-shinyaku-s-rgx-111\"><span class=\"ez-toc-section\" id=\"REGENXBIONippon_Shinyakus_RGX-111\"><\/span><strong>REGENXBIO\/Nippon Shinyaku\u2019s RGX-111<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>RGX-111<\/strong> is an investigational <strong>gene therapy<\/strong> designed to treat MPS I, a rare genetic disorder caused by a deficiency of the IDUA enzyme. It works by delivering a functional copy of the IDUA gene directly to the central nervous system (CNS), enabling brain cells to produce the enzyme. This approach aims to slow or prevent cognitive decline and neurological damage associated with MPS I.<\/p>\n\n\n\n<p>Interim results from an ongoing <strong>Phase I\/II trial<\/strong> have shown encouraging biological activity and a favorable safety profile. Recognized for its potential, RGX-111 has been granted <strong>Orphan Drug Designation (ODD), Rare Pediatric Disease Designation (RPDD), and Fast Track Designation (FTD)<\/strong> by the US FDA for <a href=\"https:\/\/www.delveinsight.com\/report-store\/mucopolysaccharidosis-i-market\">mucopolysaccharidosis type I treatment<\/a>.<\/p>\n\n\n\n<p>Recently, in March 2025, REGENXBIO Inc. finalized its previously announced strategic partnership with Nippon Shinyaku. Under this agreement, the two companies will collaborate on the development and commercialization of RGX-111 for mucopolysaccharidosis type I, also known as Hurler syndrome, in both the United States and Asia.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"h-immusoft-s-isp-001\"><span class=\"ez-toc-section\" id=\"Immusofts_ISP-001\"><\/span><strong>Immusoft\u2019s ISP-001<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p><strong>ISP-001<\/strong> is an autologous <strong>B cell therapy<\/strong> engineered to produce human alpha-L-iduronidase for treating MPS I (Hurler syndrome). The drug is currently being tested in a <strong>Phase I MPS I clinical trial<\/strong>. It received <strong>Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD)<\/strong> from the US FDA in 2018 for MPS I. In January 2025, Immusoft announced encouraging results from the first human trial of ISP-001, which will be presented at the WORLDSymposium 2025 in San Diego.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-what-lies-ahead-in-mps-i-treatment\"><span class=\"ez-toc-section\" id=\"What_Lies_Ahead_in_MPS_I_Treatment\"><\/span><strong>What Lies Ahead in MPS I Treatment?<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>The future of MPS I treatment beyond ERT is promising, with advancements in gene therapy, substrate reduction therapy, and small molecule treatments offering new hope for better outcomes. While ERT, such as laronidase, has been effective in addressing some systemic symptoms, it is limited by its inability to cross the blood-brain barrier and fully address neurological complications. Future therapies aim to overcome these limitations by delivering more targeted and long-lasting effects.<\/p>\n\n\n\n<p><a href=\"https:\/\/www.delveinsight.com\/report-store\/mucopolysaccharidosis-i-market\">Gene therapy for MPS I treatment<\/a> is emerging as a transformative approach, with the potential to provide a one-time treatment that addresses the underlying genetic defect. AAV and lentiviral vectors are being explored to deliver functional copies of the defective gene, potentially enabling sustained production of the missing enzyme. Early-stage MPS I clinical trials have shown encouraging results, with reduced GAG accumulation and improved cognitive function. Additionally, gene editing techniques such as <a href=\"https:\/\/www.delveinsight.com\/report-store\/crisper-market-forecast\">CRISPR-Cas9<\/a> hold promise for precise correction of the genetic mutation, potentially offering a permanent cure.<\/p>\n\n\n\n<p>Beyond gene therapy, novel approaches such as substrate reduction therapy (SRT) and chaperone therapy are being developed to improve cellular metabolism and enhance the stability and activity of endogenous enzymes. Intrathecal and intracerebroventricular delivery methods are also being tested to enhance CNS penetration and address neurological symptoms more effectively. Combined therapies, including ERT alongside gene therapy or SRT, could provide a more comprehensive treatment strategy. These advances have the potential to significantly improve both the quality of life and life expectancy for MPS I patients.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><a href=\"https:\/\/www.delveinsight.com\/report-store\/mucopolysaccharidosis-i-market\"><img decoding=\"async\" width=\"1024\" height=\"194\" src=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155329\/Mucopolysaccharidosis-Type-I-Market-Outlook--1024x194.png\" alt=\"Mucopolysaccharidosis-Type-I-Market-Outlook-\" class=\"wp-image-31334\" srcset=\"https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155329\/Mucopolysaccharidosis-Type-I-Market-Outlook--1024x194.png 1024w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155329\/Mucopolysaccharidosis-Type-I-Market-Outlook--300x57.png 300w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155329\/Mucopolysaccharidosis-Type-I-Market-Outlook--150x28.png 150w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155329\/Mucopolysaccharidosis-Type-I-Market-Outlook--768x145.png 768w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155329\/Mucopolysaccharidosis-Type-I-Market-Outlook--1536x291.png 1536w, https:\/\/assets.delveinsight.com\/blog\/wp-content\/uploads\/2025\/03\/20155329\/Mucopolysaccharidosis-Type-I-Market-Outlook-.png 1584w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><\/a><\/figure>\n","protected":false},"excerpt":{"rendered":"<p>MPS I treatment typically involves Hematopoietic Stem Cell Transplantation (HSCT), Enzyme Replacement Therapy (ERT), or a combination of both, along with supportive care such as pain management, anti-inflammatory medications, oxygen therapy, and surgery. While these MPS I treatments help manage symptoms and improve survival, they cannot reverse existing damage. Early intervention is essential to achieve [&hellip;]<\/p>\n","protected":false},"author":14,"featured_media":31342,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[17],"tags":[22494,22492,22493,22495,22491,22496],"industry":[17225],"therapeutic_areas":[17240,17234],"class_list":["post-31340","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-articles","tag-aldurazyme","tag-mps-i","tag-mps-i-treatment","tag-mps-i-treatment-pipeline","tag-mucopolysaccharidosis-type-i","tag-mucopolysaccharidosis-type-i-treatment","industry-pharmaceutical","therapeutic_areas-endocrinology-and-metabolic-disorders","therapeutic_areas-rare-diseases"],"acf":[],"yoast_head":"<!-- This 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