Myrtelle

is a gene therapy company focused on providing hope and progress by developing transformative treatments for neurodegenerative diseases that affect patients who have few, if any, treatment options. Our lead program is a novel investigational gene therapy treatment for Canavan disease (CD), a fatal genetic brain disorder in which mutations in the ASPA gene prevent the normal expression of Aspartoac

ylase, a critical enzyme produced in oligodendrocyte cells that breaks down the neurochemical N-Acetylaspartic Acid (NAA). CD patients are affected at birth but may appear normal until several months, when symptoms begin to develop. Poor head control, abnormally large head size, eye tracking difficulty, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones such as rolling, sitting, and walking are typical. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge, with most affected children developing life-threatening complications by ten years of age. Currently, there is no cure for CD and only palliative treatments are available. Myrtelle’s social media channels are places for community members to connect and learn about Myrtelle’s initiatives. Discussion of medical and health topics should never be construed as medical advice. Contact a healthcare professional in your area for personal medical advice. Myrtelle does not represent that the information on our social media channels is accurate, complete, reliable, useful, timely, or current and Myrtelle does not undertake an obligation to update any such information. Myrtelle does not endorse any opinions or statements expressed by others on our social media channels. Myrtelle does not expressly endorse opinions or statements expressed by individuals, businesses, or channels we may ‘follow’, ‘like’, or ‘share’. At times, Myrtelle may link to external websites. Myrtelle is not responsible for the content or policies of these websites. Content submitted to our social media channels is public and will not place Myrtelle under any obligation to you.