20 Jul Papillon Therapeutics
Carter Cliff, CEO
Oct. 7 | 9:30am | FLW Ballroom F
San Diego, CA
(Private)
Papillon Therapeutics is a clinical stage biotechnology company advancing multi-systemic genetic medicines for diseases with neurologic, cardiac, and other tissue pathologies. Degenerative diseases impact multiple tissues throughout the body, inspiring our unique approach to deliver therapeutic protein to diseased tissues via an innate system within the body. 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. Published studies by scientific founder, Dr. Cherqui, demonstrate multi-systemic engraftment and functional rescue utilizing our gene modified HSPCs for Friedreich’s ataxia, Danon disease, and Alzheimer’s disease. 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.
