{"id":33690,"date":"2026-03-23T10:13:47","date_gmt":"2026-03-23T14:13:47","guid":{"rendered":"https:\/\/genomequebec.com\/?post_type=financed-project&#038;p=33690"},"modified":"2026-03-23T10:13:47","modified_gmt":"2026-03-23T14:13:47","slug":"towards-the-commercialization-of-an-omics-guided-rna-based-therapy-for-neurofibromatosis","status":"publish","type":"financed-project","link":"https:\/\/genomequebec.com\/en\/funded-projects\/towards-the-commercialization-of-an-omics-guided-rna-based-therapy-for-neurofibromatosis\/","title":{"rendered":"Towards the commercialization of an omics-guided RNA-based therapy for neurofibromatosis"},"content":{"rendered":"\n<p><strong>User:<\/strong> RNA4RARE Therapeutics Inc.<\/p>\n\n\n\n<p>Neurofibromatosis (NF) is a rare genetic condition (1 in 2000) that causes tumors to develop from glial cells in the nervous system. It occurs when these glial cells lose protective proteins that regulate normal cell growth\u2014neurofibromin in NF1 and merlin in NF2\u2014allowing tumors to form. This leads to diverse symptoms, such as hearing and vision loss, nerve pain, learning and motor disabilities, and reduced quality of life. Treatment options are limited: available medications for NF1 can slow tumor growth but do not replace the missing protein, and no approved drug therapies exist for NF2. As a result, many patients rely on long-term monitoring, surgery, or radiation, and tumors often recur.<\/p>\n\n\n\n<p>This co-led academic-industry project aims to address the underlying protein deficiency in NF by using therapeutic messenger RNA (mRNA) to provide cells with instructions to produce the missing NF proteins. The mRNA is delivered using lipid nanoparticles, which protect mRNA and enable targeted cellular delivery. <\/p>\n\n\n\n<p>The project will focus on optimizing mRNA design for clinical development. Using advanced omics-based approaches, the team will engineer mRNA constructs to prolong protein expression, enhance protein production in target cells, and limit activity in non-target tissues. The collaboration combines RNA4RARE\u2019s expertise in mRNA design and delivery, Dr. Stratton\u2019s leadership in omics and human cell and tissue analysis, and McGill\u2019s strong clinical research ecosystem. &nbsp;The Neuro is internationally recognized in neuroscience, open science, translational platforms, and early-phase clinical research, including pioneering RNA therapy trials for rare neurological disorders. &nbsp;If successful, this work could lead to the first targeted therapies for NF and establish a broader platform for treating other rare glial diseases caused by loss of function variants.<\/p>\n","protected":false},"featured_media":0,"template":"","class_list":["post-33690","financed-project","type-financed-project","status-publish","hentry"],"acf":[],"_links":{"self":[{"href":"https:\/\/genomequebec.com\/en\/wp-json\/wp\/v2\/financed-project\/33690","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/genomequebec.com\/en\/wp-json\/wp\/v2\/financed-project"}],"about":[{"href":"https:\/\/genomequebec.com\/en\/wp-json\/wp\/v2\/types\/financed-project"}],"wp:attachment":[{"href":"https:\/\/genomequebec.com\/en\/wp-json\/wp\/v2\/media?parent=33690"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}