Papillon Therapeutics is advancing gene therapy treatments for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease, and Alzheimer’s disease. We specialize in developing gene modified hematopoietic stem and progenitor cell (HSPC) therapies, leveraging the natural capacity of these unique human cells to safely deliver a gene therapy throughout the body. Our clinical stage program, an HSPC therapy for treatment of Cystinosis, reported positive data in a Phase 1/2 clinical trial and is being advanced by Novartis. Building on this success, we are advancing gene modified HSPC treatments for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease, and Alzheimer’s disease. Non-dilutive funding awarded by the California Institute of Regenerative Medicine (CIRM) to our scientific founders totals $28.7 million towards the Cystinosis, Friedreich's ataxia, and Danon disease programs.