Funding outcome

The Problem Achondroplasia is the most common cause of dwarfism. It is caused by a genetic mutation in the FGFR3 protein, which sends an abnormal signal to bone growth plates. This prevents cartilage from converting into bone, leading to short stature and lifelong health issues, such as spinal complications and the need for C-sections during childbirth. The current treatment, vosoritide, can restart bone growth but costs over $400,000 CAD per year and requires painful daily injections. Due to this high cost and treatment burden, many families cannot access it. The Solution: A Muscle 'Bio-Factory' We are developing a way for the body to produce its own medicine. Using self-amplifying RNA (saRNA), we provide the body with instructions to produce the same growth-promoting protein found in vosoritide. By injecting saRNA into the muscle, we turn it into a 'local factory' that continuously releases the medicine into the bloodstream. Unlike regular mRNA, saRNA copies itself inside cells, meaning a single low-dose injection could last much longer. The Challenge The immune system often mistakes RNA instructions for a virus and shuts them down. To solve this, we engineered several versions of saRNA. Our lead candidate, Set 4, is modified to 'sneak past' immune defenses while producing high levels of the medicine. Our Research Plan In this project, we will: Test in mice: Identify which saRNA version lasts longest with the least inflammation. Verify the medicine: Ensure our saRNA-produced protein is as effective as vosoritide. Prove the concept: Test in bone cells to confirm it successfully triggers the 'grow' signal. The Goal This study provides the evidence needed for a cheaper, more accessible treatment for achondroplasia, potentially replacing daily injections with long-lasting, affordable care.