Company Description: Papillon Therapeutics is a biotechnology company advancing genetic medicines for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease, and Alzheimer’s disease. Inherited conditions often cause degeneration of multiple tissues throughout the body, inspring our approach to develop therapies that offer patients lifelong multi-systemic therapeutic benefit from a single treatment. Gene modified hematopoietic stem and progenitor cell (HSPC) therapy is a commercially proven approach for disease modifying and potentially curative treatments in certain inherited disorders. 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. 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.