PPL-001

PPL-001 is an experimental gene-edited hematopoietic stem and progenitor cell (HSPC) therapy developed by Papillon Therapeutics to treat Friedreich’s ataxia, a rare inherited neurodegenerative disorder. The therapy utilizes gene editing, specifically targeted excision, to correct the GAA repeat expansion mutation in the FXN gene (frataxin), which is responsible for most cases of Friedreich’s ataxia. By correcting this mutation in patient-derived CD34+ HSPCs, PPL-001 aims to restore frataxin protein production across multiple organ systems, including the heart, brain, and spinal cord. This multi-systemic approach is designed to slow or reverse disease progression by addressing the underlying genetic defect. PPL-001 has received both Orphan Drug and Rare Pediatric Disease designations from the FDA and is currently in IND-enabling studies.