Building New Regulatory & Funding Pathways for Ultra-Rare Kidney Therapies to Bring Bespoke Genetic Medicines into the Clinic
2:30 pm - Wednesday 17th March 2027
- Reframing development models for ultra-rare therapies where conventional commercial economics prevent promising programs from progressing
- Evaluating conditional, investigational and continuously monitored approval pathways that could reduce barriers between proof-of-concept and patient treatment
- Coordinating regulators, academia, funders, payers and industry to create sustainable routes for developing therapies for ultra-small genetic populations at scale