Claire Clelland (MD, PhD)

Dr. Clelland is building what every C9 carrier hopes for: a one-time fix at the genomic level. Her CRISPR work has shown the most complete reversal of C9 pathology demonstrated to date in patient-derived neurons. She is now partnering with Denali Therapeutics to solve the hardest remaining problem: getting that edit into the brain via a simple IV infusion. A project she is not yet public about is her work screening and optimizing gene therapy for the CNS using brain-dead trauma patients. This use of humans to improve gene therapy in a compartment nobody has successfully targeted is nearly unparalleled in utility for C9 and other neurological diseases. The cost for a first-in-human dose is ~$10M. This is one of the projects that should be proposed for funding by CureC9. Dr. Clelland chose C9orf72 because her patients have FTD. The most common cause of familial FTD is the C9 repeat expansion, so to cure FTD she needs to cure C9. Beyond the science, she brings a focus on translation that keeps every conversation grounded in what actually matters: getting a therapy to patients.