Papillon is dedicated to delivering cures from within by harnessing the body’s own cells. Overcoming the targeting barriers of traditional genetic medicines, the company’s proprietary platform utilizes patient-derived hematopoietic stem cells to systemically distribute functional proteins across multiple aff ected organs. Papillon’s lead program, PPL-001, is a one-time, potentially lifetime-durable treatment designed to arrest the progression of Friedreich’s ataxia across aff ected tissues—including the brain, spine, heart, and pancreas. Validated by a landmark clinical partnership with Novartis, Papillon is advancing a pipeline of transformative cell therapies engineered to restore health across a lifetime.

Papillon Therapeutics

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Papillon Therapeutics

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