Papillon Therapeutics is a clinical stage biotechnology company advancing multi-systemic genetic medicines for diseases with neurologic, cardiac, and other tissue pathologies. Our clinical stage program, a gene modified hematopoietic stem and progenitor cell (HSPC) therapy for treatment of Cystinosis, reported positive data in a Phase 1/2 clinical trial and is being advanced by Novartis. Funding awarded by the California Institute of Regenerative Medicine (CIRM) and NIH to our scientific founders totals over $30 million towards our Cystinosis, Friedreich’s ataxia, and Danon disease programs.