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.

Link to Papillon Therapeutics

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.

News and Press Releases

| Jan 23, 2023

A Gene-Edited Cell Therapy for an Incurable Disease: Researchers Receive USD 4.8M Grant to Treat Friedreich’s Ataxia

Industry News