{"id":6548,"date":"2025-06-18T09:56:59","date_gmt":"2025-06-18T13:56:59","guid":{"rendered":"https:\/\/cqdm.org\/?post_type=project&#038;p=6548"},"modified":"2025-06-18T10:01:10","modified_gmt":"2025-06-18T14:01:10","slug":"a-brain-specific-approach-to-rna-therapeutics-developing-a-novel-protein-delivery-strategy-for-the-central-nervous-system","status":"publish","type":"project","link":"https:\/\/cqdm.org\/en\/achievement\/funded-projects\/a-brain-specific-approach-to-rna-therapeutics-developing-a-novel-protein-delivery-strategy-for-the-central-nervous-system\/","title":{"rendered":"A brain-specific approach to RNA therapeutics: developing a novel protein delivery strategy for the central nervous system"},"content":{"rendered":"\n<div class=\"wp-block-columns is-layout-flex wp-container-core-columns-is-layout-28f84493 wp-block-columns-is-layout-flex\">\n<div class=\"wp-block-column is-layout-flow wp-block-column-is-layout-flow\">\n<p class=\"is-style-suptitle\"><strong>Principal Investigator<\/strong><\/p>\n\n\n\n<p><strong>S\u00e9bastien Tremblay<\/strong><br>Universit\u00e9 Laval &#8211; CERVO<\/p>\n<\/div>\n\n\n\n<div class=\"wp-block-column is-layout-flow wp-block-column-is-layout-flow\">\n<p class=\"is-style-suptitle\"><strong>Co-investigator<\/strong><\/p>\n\n\n\n<p><span style=\"\"><b>Brian MacVicar<br><\/b><\/span>University of British Columbia<\/p>\n<\/div>\n<\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\"><span style=\"\">Project of <\/span><strong>$1,464,765<strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong><strong> over 3 years<\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list is-style-checkmark\">\n<li><strong><strong>Supported by CQDM through:<\/strong><\/strong><br>Minist\u00e8re de l\u2019\u00c9conomie, de l\u2019Innovation et de l\u2019\u00c9nergie du Qu\u00e9bec (MEIE)<\/li>\n\n\n\n<li><strong><strong><strong>And by co-funding partners:<\/strong><\/strong><\/strong><br>&#8211; RNA Technologies &amp; Therapeutics (RNA T&amp;T)<br>&#8211; CereCura Nanotherapeutics<br>&#8211; Brain Canada Foundation<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>Challenge<\/strong><\/h3>\n\n\n\n<p>Delivery of therapeutic proteins to the brain has been a longstanding barrier to Central Nervous System (CNS) drug development. Protein replacement or supplementation strategies hold promise for multiple brain diseases, from enzyme-replacement for neuronopathic lysosomal disorders, to growth factor supplementation for neurodegeneration, to cytokine immunotherapy for brain cancers. However, most brain diseases remain poorly treated in part due to the inability to deliver proteins to the brain with a disease-appropriate biodistribution.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>Solution<\/strong><\/h3>\n\n\n\n<p>Prof. S\u00e9bastien Tremblay\u2019s team (Universit\u00e9 Laval-CERVO), in collaboration with Prof. Brian MacVicar\u2019s team (University of British Columbia; UBC) will address the brain protein delivery challenge using lipid nanoparticle (LNP) to bring to brain cells the RNAm blueprint that encodes for a therapeutic protein. LNP-RNAm products instruct the host cell to secrete a desired protein that then diffuses broadly throughout the brain, effectively harnessing brain cells to become \u2018protein factories\u2019 within the brain itself.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>Expected Achievements\/Impact:<\/strong><\/h3>\n\n\n\n<p>The co-funders <a href=\"https:\/\/www.cerecura.com\/\" target=\"_blank\" rel=\"noopener\">CereCura Nanotherapeutics Inc.<\/a> and <a href=\"https:\/\/www.rnatechnologies.com\/\" target=\"_blank\" rel=\"noopener\">RNA Technologies and Therapeutics Inc<\/a>. have shown the strategy is viable in rodents, demonstrating this versatile technology can deliver multiple proteins with unprecedented spatiotemporal distribution in the brain. Leveraging the expertise of Prof. S\u00e9bastien Tremblay and his Translational Neurosurgery Laboratory in optimizing genetic drug delivery to non-human primate (NHP) brains and that of Prof Brian MacVicar at UBC in mouse models of neurological diseases,&nbsp;&nbsp; this multidisciplinary team&nbsp; will 1) demonstrate the feasibility of LNP-RNA-induced protein delivery to NHP brain, 2) optimize the RNA cargo of a lead candidate drug product using in vitro models and 3) validate its therapeutic in a specific disease use case (neuronopathic lysosomal disease).<\/p>\n\n\n\n<p>Moreover, the combination of Montreal-based company RNA technologies and Therapeutics&#8217; expertise in RNA sequence design and optimization, high-quality RNA synthesis (preclinical and clinical), lipid formulation development and LNP surface modification for specific targeting, with the expertise of Vancouver LNP development hub, will bring us one step closer to first-in-human trials, bringing hope to Quebecers and Canadians suffering from debilitating brain diseases.<\/p>\n","protected":false},"featured_media":3830,"template":"","project-category":[96],"class_list":["post-6548","project","type-project","status-publish","has-post-thumbnail","hentry","project-category-small-molecules"],"acf":[],"_links":{"self":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project\/6548","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project"}],"about":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/types\/project"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media\/3830"}],"wp:attachment":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media?parent=6548"}],"wp:term":[{"taxonomy":"project-category","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project-category?post=6548"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}