# Papillon Therapeutics > Papillon Therapeutics harnesses the body's own cells for natural in vivo delivery of one-time treatments for life-threatening genetic diseases. Papillon's lead program PPL-001 is a preclinical treatment candidate for Friedreich's ataxia, built on a clinically validated platform with published Phase I/II results in cystinosis (NEJM, 2026). ## Pages - [Home](/): Company overview, pipeline, science, team, and news. - [Contact](/contact): Partner and general inquiries. ## Optional - [Privacy Policy](/privacy): How we handle visitor information. - [Terms of Service](/terms): Website terms of use.