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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 Ph1/2 clinical stage program that recently reported positive data for a gene-modified HSPC therapeutic candidate, and multiple preclinical stage programs that benefit from this clinical program.

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