Papillon Therapeutics is a biotechnology company advancing new therapies tailored to address lysosomal and mitochondrial dysfunctions that underly life-threating inherited conditions, such as Friedreich's ataxia. We specialize in developing gene-modified hematopoietic stem and progenitor cell (HSPC) therapies, leveraging the natural capacity of these unique human cells to perform multi-systemic repair of damaged tissues. Our pipeline includes a partnered, fully enrolled clinical stage program to assess safety and efficacy of a gene-modified HSPC therapeutic candidate, and multiple preclinical stage programs that benefit from the clinical program.
