RNA4RARE Therapeutics Marks First Anniversary and Advances Neurofibromatosis type 1 and 2 mRNA Programs Through a $900,000 Research Project Supported by Génome Québec/Genome Canada
MONTRÉAL, September 15, 2026 — RNA4RARE Therapeutics Inc., a Canadian biotechnology company developing disease-modifying mRNA therapeutics for rare diseases, today marks its first anniversary and announces the advancement of its lead programs through a $900,000 research project supported by Génome Québec/Genome Canada, in collaboration with Jo Anne Stratton and her team at The Neuro (Montreal Neurological Institute-Hospital) of McGill University.
Founded in September 2025 by scientists and patient advocates, RNA4RARE was created with a clear mission: to advance RNA therapeutics for people living with rare diseases for whom treatment options remain limited or unavailable. Over its first year, the company has established its scientific and research foundations, built collaborations with leading academic and clinical experts, and advanced the development of its mRNA therapeutic approach.
“Our first year has been focused on building the scientific, translational and collaborative foundation needed to move RNA4RARE forward,” said Dr. Michael Zimmer, Co-Founder and Chief Scientist Officer at RNA4RARE Therapeutics. “The support from Génome Québec and Genome Canada, together with our collaboration with Prof. Stratton and The Neuro, will enable us to generate important proof-of-concept data and address key scientific questions as we advance our initial therapeutic programs. We are grateful to our collaborators and partners who share our commitment to developing new therapeutic possibilities for people living with rare diseases.”
RNA4RARE's lead programs are focused on neurofibromatosis (NF), a group of rare genetic disorders in which loss or dysfunction of key tumor-suppressor proteins can lead to the development of tumors and significant lifelong complications. The company'sapproach is based on mRNA replacement therapy, with the goal of restoring the production of proteins that are deficient or absent as a result of disease-causing genetic mutations.
A central challenge in developing mRNA therapeutics for diseases beyond the liver is achieving efficient and selective delivery to the relevant disease-associated cells and tissues. RNA4RARE is developing an mRNA delivery approach designed to enable delivery beyond the liver, combined with tissue-specific microRNA-based control mechanisms intended to help regulate where therapeutic protein expression occurs. The company is initially applying this approach to neurofibromatosis types 1 and 2 with the goal of establishing proof of concept for a broader RNA therapeutic platform.
“RNA4RARE is building on years of rigorous research and a strong scientific foundation,” said Dr. Guy Rouleau, Chair of RNA4RARE’s Scientific Advisory Board and Director of the Department of Neurology and Neurosurgery at McGill University. “Seeing discoveries we helped uncover progress toward potential therapies for rare diseases is particularly meaningful, and I am proud to contribute to their continued development.”
Through the research collaboration with Jo Anne Stratton, a principal investigator at The Neuro and assistant professor at McGill University, RNA4RARE and her team are working to generate the preclinical evidence needed to evaluate and advance the therapeutic approach.
“This collaboration allows us to address important scientific questions at a critical stage of development and generate the data needed to guide the programs forward,” said Prof. Stratton. “Our work is focused on building the evidence needed to translate an innovative mRNA approach into a potential therapeutic strategy for neurofibromatosis.”
This $900,000 research project is supported by $600,000 in funding from Génome Québec/Genome Canada through the Genomic Application Partnership Program (GAPP), with a $300,000 contribution from RNA4RARE Therapeutics.
For Stéphanie Lord-Fontaine, Vice President, Scientific Affairs at Génome Québec, this project is perfectly aligned with the objectives of the GAPP program. It is an excellent example of academic and industry collaboration aimed at leveraging genomics to accelerate the commercialization of therapeutic options for the benefit of affected individuals.
As RNA4RARE enters its second year, the company will focus on advancing its lead neurofibromatosis programs, generating key proof-of-concept data, and continuing to build the scientific, clinical and patient-focused partnerships needed to move promising RNA therapeutic approaches closer to patients.
About RNA4RARE Therapeutics
RNA4RARE Therapeutics Inc. is a Canadian biotechnology company dedicated to accelerating the development of mRNA-based therapies for rare diseases. Founded by a team of scientists and patient advocates, RNA4RARE is driven by a shared mission to bridge the gap between scientific discovery and accessible treatments. Through a collaborative model that brings together industry and academic research, clinical expertise, patient lived experience, and strategic partnerships, RNA4RARE aims to advance innovative RNA-based therapies for rare diseases by developing approaches that enable mRNA to reach disease-relevant cells and tissues beyond the liver, with the goal of providing new therapeutic options for patients with limited or no effective treatments.
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