About Event
Welcome to the 2nd Rare & Genetic Kidney Disease Drug Development Summit
Putting a spotlight on exciting pathways for therapeutic intervention, recapitulating genetic mutations with robust preclinical models, and investigating innovative trial design to evaluate safety and efficacy, the Rare & Genetic Kidney Disease Drug Development Summit is uniquely positioned to provide strategic insight into paving the way to regulatory approval. Hear from industry experts establishing a clinical pathway to correlate novel biomarkers with clinical outcome and leading the charge in designing safe interventional trials in the paediatric population to bring effective therapies to patients.
Unmissable highlights for 2022:
Forge a deeper understanding of genetic drivers of disease and translate new insights into underlying pathophysiology to reclassify disease subtypes with Goldfinch Bio, Harvard Medical School, and Washington University to investigate exciting opportunities for therapeutic intervention
Harness efficient patient recruitment of cohorts with specific disease criteria and enhance enrolment of underrepresented patient populations with Takeda, Nephcure, and Otsuka to bridge the gap between patients and trials and leverage patient registries to recruit adults and children
Define the next step in developing and validating early biomarkers of therapeutic efficacy and investigate disease specific endpoint development with the FDA and Sanofi to identify patients at risk of rapid progression and leverage surrogate endpoints to attain regulatory approval
Navigate exciting progress in gene therapy, small molecule, microRNA and complement targeted therapies with Regulus Therapeutics, Visterra, and Purespring Therapeutics, to enlighten opportunities for therapeutic intervention
Investigate adaptive, basket, and open label trials with untraditional master protocols and enriched patient populations with Travere, Chinook Therapeutics, and Vifor Pharma to facilitate clinical trial efficiency with reduced heterogeneity