RNA medicines—short strands of nucleic acids that can turn genes “up” or “down”—have changed how we treat brain diseases. For example, nusinersen for spinal muscular atrophy and tofersen for ALS are given by spinal injection to reach the brain and spinal cord. These breakthroughs show that RNA drugs work in the nervous system. However, before these treatments can reach patients, they must be tested in living systems to ensure safety and effectiveness. The goal of PRIME‑RNA is to help researchers turn promising RNA drug ideas into therapies by building an in vivo testing platform at the IRCM. This platform will test how RNA drugs are best delivered to the brain, whether they engage the right targets, how well they work in disease models, and whether they are safe in mice before moving toward clinical trials. PRIME-RNA is integrated with the IRCM RNA Therapeutics core facility, which focuses on RNA design and synthesis, creating a complete pipeline from molecule design to preclinical validation. It is also part of the Sidney-Altman RNA Therapeutics Hub, an IRCM-led initiative that leverages the expertise of RNA researchers across a network of research centres, hospitals, and industry partners. This means researchers can move their discoveries forward faster, at lower cost, and without relying on international facilities. PRIME‑RNA will be accessible to public and private partners across Québec and Canada through a transparent and equitable process. With support from Brain Canada and sponsors, the platform will accelerate the development of innovative RNA-based treatments for brain cancers and neurological diseases, areas of high unmet medical need, improve patient outcomes, train the next generation of scientists in RNA therapeutics to build expertise in this rapidly growing field, while strengthening Canada’s position as a leader in brain health research.
