20 Jul Papillon Therapeutics
Carter Cliff, CEO
Oct. 7 | 12:00pm | FLW Ballroom G
San Diego, CA
(Private)
Papillon Therapeutics is a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease. We are advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease, targeting neurodegenerative 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 and is now licensed to Novartis who are advancing “DFT383 in Pediatric Participants With Nephropathic Cystinosis”. Our lead preclinical stage program targeting Friedreich’s ataxia (PPL-001) aims to submit IND within a year and has received funding from CIRM. Our second preclinical stage program targeting Danon disease (PPL-002) will hold Pre-IND this year and received funding from CIRM.
