Rare diseases collectively affect more than 300 million people worldwide, yet fewer than 5% have an approved treatment. As cell and gene therapies (CGTs) progress from scientific aspiration to commercial and clinical reality, rare and ultra‑rare diseases remain at the center of the field’s most significant advances. This session brings together leaders representing biotech, regulatory agencies, payer organizations, investor communities, and patient advocacy to explore how CGTs are realistically being developed, delivered, and accessed today, with a focus on the latest clinical data shaping this next era. Panelists will examine real‑world evidence emerging from 2025–2026 clinical programs and discuss how adaptive trial strategies, single‑arm designs, and alternative data sources are enabling robust evidence generation despite small patient populations.