10 May 16:00-18:00
This practical and educational session will deliver an introduction and overview on the use of evolving gene therapies in the rare disease space and the challenges faced in clinical development, including discussion on use of biomarkers, novel complex endpoints and innovative trial designs. Insights will be provided into ‘lessons learnt’ from the perspectives of four highly experienced experts in the field.
12 May 16:30-18:00
This panel discussion will provide insights on the specific challenges with patient engagement in gene therapy and rare diseases. Our panel of expert speakers include leading patient advocates, leaders in rare disease gene therapy and drug development, and experts in market access for patients with rare diseases.