Workshop A: Integrating Advanced Disease Models to Improve Translational Predictability in Rare Kidney Disease Drug Discovery

Join this interactive workshop to explore how researchers are improving the translational success of rare kidney disease therapeutics by integrating human datasets, advanced disease models, and next-generation molecular profiling approaches. Through collaborative discussion, participants will examine how emerging technologies and modeling strategies can better predict therapeutic efficacy and bridge the gap between preclinical discovery and clinical development.

This workshop will gather experts to discuss:

  • Integrating human genomic and molecular datasets with preclinical models to improve target validation
  • Leveraging organoid systems, iPSC-derived models, and mutation-specific animal models to better reflect patient disease biology
  • Applying multi-omics approaches and spatial profiling to identify actionable therapeutic targets
  • Improving predictive preclinical assays that can better forecast clinical efficacy and reduce late-stage failure
  • Evaluating challenges in translating genetic discoveries into druggable targets
  • Aligning preclinical evidence packages with clinical development strategies