We will explore:
- Why ultra-rare diseases challenge traditional biotech and commercial models
- How non-profit, mission-driven approaches can enable therapy development
- Key “access-by-design” considerations: regulatory strategy, reimbursement, and cross-border care
- The role of real-world evidence, registries, and manufacturing continuity
- Lessons from pioneering therapies such as Strimvelis and Waskyra
- How European policy is shaping access to ATMPs
Ultra-rare diseases often fall outside conventional pathways — but new models are emerging. This session will provide practical insights for researchers, clinicians, policymakers, and innovators working to bring transformative therapies to patients who need them most.
Registration HERE
More information to the attached flyer.
