RIAAN RESEARCH INITIATIVE INC
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Mission Statement
Riaan Research Initiative is dedicated to advancing, accelerating, and funding translational scientific research to generate treatments for severe and lethal neurodegenerative genetic disorders that impact the pediatric population. Our signature program is developing the world's first gene therapy for Cockayne syndrome, a devastating DNA repair and transcription neurodegenerative genetic disorder that is almost always fatal in children. After successfully completing proof-of-concept studies and engaging the FDA, we are now in the process of toxicology studies, and manufacturing the AAV9 therapy alongside our collaborators at UMass Chan Medical School, Andelyn Biosciences, and Charles River Labs. Next stop, clinical trials! Help us raise the needed funds (one million dollars) to support administering this therapy to the first set of children, including covering hospital stays, imaging/tests, medications, travel costs, and short-term follow up. This entire project has been funded by supporters, and the community. Thank you.