PPL-001
PPL-001 is a gene modified hematopoietic stem and progenitor cell (HSPC) designed to correct the mutation located in the frataxin (FXN) gene of patients with Friedreich’s ataxia. Patients receiving gene modified HSPC treatments undergo a standard procedure of HSPC mobilization and collection, followed by gene modification, cell processing, and infusion.
Stages of Development for PPL-001
The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.
Syngeneic hematopoietic stem and progenitor cell (HSPC) transplantation prevented neurodegeneration in the YG8R FA mouse model. A second study showed autologous HSPC transplantation using the CRISPR-Cas9 system to edit the cells and remove the GAA repeats, restored frataxin expression and normalizing mitochondrial activity.
The gene-editing approach in HSPCs isolated from healthy and FA patients’ peripheral blood demonstrated normal hematopoiesis of gene-edited cells in vitro and in vivo.
PPL-001 is a gene modified hematopoietic stem and progenitor cell (HSPC) therapy. Published preclinical studies by scientific founder, Dr. Stephanie Cherqui, demonstrated multi-systemic engraftment and functional rescue in gene modified HSPCs from Friedreich’s ataxia patients.
PPL-001, currently in IND enabling studies, is a gene modified hematopoietic stem and progenitor cell (HSPC) designed to correct the mutation located in the FXN gene of patients with Friedreich’s ataxia.
Papillon’s candidate PPL-001 has been granted Rare Pediatric Disease designation by the FDA. PPL-001 previously received Orphan Drug designation. The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that affect individuals under the age of 18.