{"id":8987,"date":"2026-11-09T10:46:06","date_gmt":"2026-11-09T15:46:06","guid":{"rendered":"https:\/\/cqdm.org\/?post_type=project&#038;p=8987"},"modified":"2026-11-09T10:50:18","modified_gmt":"2026-11-09T15:50:18","slug":"engineering-nanocarriers-for-rna-delivery-to-schwann-and-meningeal-cells-to-enable-rna-therapeutics-for-neurofibromatosis","status":"publish","type":"project","link":"https:\/\/cqdm.org\/en\/achievement\/funded-projects\/engineering-nanocarriers-for-rna-delivery-to-schwann-and-meningeal-cells-to-enable-rna-therapeutics-for-neurofibromatosis\/","title":{"rendered":"Engineering Nanocarriers for RNA Delivery to Schwann and Meningeal Cells to Enable RNA Therapeutics for Neurofibromatosis"},"content":{"rendered":"\n<div class=\"wp-block-columns is-layout-flex wp-container-core-columns-is-layout-7387b849 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 wp-block-paragraph\"><strong>Principal Entity:<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">RNA4RARE Therapeutics Inc<\/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\"><strong>Project of $400,000 over 1 years<\/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 du D\u00e9veloppement \u00e9conomique, de l\u2019Innovation et de l\u2019\u00c9nergie (MDEIE)<\/li>\n\n\n\n<li><strong><strong>And by co-funding partner:<\/strong><\/strong><br>&#8211; RNA4RARE Therapeutics Inc<\/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>The project in detail:<\/strong><\/h3>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>Challenge:<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Neurofibromatosis types 1 and 2 (NF1 and NF2) are genetic disorders that can cause tumors to develop along peripheral nerves and within the central nervous system. Current treatments generally manage tumors or downstream signaling rather than restoring the function of the affected NF1 or NF2 genes. A major barrier to developing genetic therapies is the safe and efficient delivery of therapeutic cargo to disease-relevant cells, particularly Schwann and meningeal cells. There is a critical need for delivery technologies capable of transporting genetic therapies to these disease-relevant cells safely and effectively.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Solution:<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">This project, led by RNA4RARE Therapeutics Inc., develops and evaluates a new generation of delivery systems for genetic therapies. The team will engineer lipid nanoparticles (LNPs) and explore other emerging technologies designed to transport therapeutic mRNA to Schwann, meningeal, and other disease-relevant cells, with the goal of achieving effective extrahepatic delivery. Using high-throughput laboratory screening, comparative formulation studies, and in vivo validation in mouse models, the team will identify lead delivery technologies that achieve efficient cellular uptake, therapeutically meaningful protein expression, and appropriate preliminary safety and tolerability. The approach will also investigate the formulation characteristics associated with delivery to disease-relevant tissues and cells and support the rational design of future delivery systems.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Expected Achievements \/ Impacts:<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">The project is expected to generate and validate lead delivery technologies capable of effectively reaching the cells involved in NF1 and NF2, laying the foundation for future therapies designed to restore NF1 or NF2 function. The resulting knowledge will advance RNA and gene therapy development for neurological and rare diseases. By advancing a platform developed by Quebec-based RNA4RARE, the project will also strengthen Quebec\u2019s expertise and innovation capacity in advanced genetic medicine and nanomedicine technologies.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>\n","protected":false},"featured_media":3823,"template":"","project-category":[97],"class_list":["post-8987","project","type-project","status-publish","has-post-thumbnail","hentry","project-category-biological-and-therapeutic"],"acf":[],"_links":{"self":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project\/8987","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\/3823"}],"wp:attachment":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media?parent=8987"}],"wp:term":[{"taxonomy":"project-category","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project-category?post=8987"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}