Rarity PBC

Rarity PBC is a public benefit corporation dedicated to making life-saving gene therapies accessible to those affected by rare genetic diseases. We believe that every patient, regardless of rarity, deserves access to proven, transformative treatments. By focusing on overlooked but clinically validated therapies, we aim to bridge the gap between groundbreaking research and real-world impact, giving hope to patients and families who have long been underserved. Our lead program centers on ADA-SCID lentiviral vector gene therapy, a pioneering treatment that offers a cure for patients with this severe immunodeficiency disorder. Rarity remains committed to expanding its portfolio to include additional gene therapies for other ultra-rare genetic conditions, staying true to our mission of delivering hope and new possibilities to those who need it most.