Rarity PBC: Where Mission Meets Gene Therapy

Paul Ayoub, co‑founder of Rarity PBC, joins this episode of Moving Medicine Forward to take us behind the scenes of developing sustainable, patient‑first gene therapies for rare genetic diseases.Paul explains how the guidance of influential mentors, combined with his experience in cell and gene therapy, shaped who he is and ultimately guided Rarity’s mission. This episode explores ADA‑SCID, the realities of protective isolation for families, and the potential for a one‑time gene therapy to transform children’s lives, while highlighting why resilience drives innovation in rare disease care.Rarity PBC is a mission-driven public benefit corporation focused on developing accessible and affordable gene therapies for rare diseases. Learn more by visiting their website: Rarity PBC | gene therapy 1:00 Paul introduces Rarity PBC and its mission to deliver gene therapies for rare diseases.3:00 Challenging the misconception that rare disease therapies can’t be sustainable.4:00 The scientific promise of gene therapy and the personal motivation behind the work.6:30 Why rare disease innovation can (and must) balance purpose and viability.8:00 What ADA-SCID is, why it’s urgent, and how early intervention saves lives.9:40 The isolation, vigilance, and emotional toll of living in a protective bubble.10:45 A story that highlights what “normal life” truly means for families.12:00 A one-time, autologous gene therapy that addresses the root genetic cause.13:45 Why gene therapy represents a shift toward curative, value-based care.14:00 How Rarity’s structure embeds patient-first decision-making.17:00 Why expertise, continuity, and culture matter in gene therapy development.18:20 Long-term outcomes, manufacturing scale-up, and the path to FDA approval.