Explore the Agenda
7:00 am Check In, Coffee & Light Breakfast
8:00 am 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
10:30 am Morning Refreshments
11:00 am Workshop B: Bridging the Phase II Endpoint Gap in Rare Kidney Disease Trials by Validating Surrogate Biomarkers Across Diseases & Data Platforms to Enable Earlier Proof-of-Concept & Accelerate Drug Development
Identifying reliable Phase II endpoints remains one of the biggest barriers to accelerating drug development in rare kidney diseases. While hard outcomes such as dialysis or kidney failure are well established for late-stage trials, earlier signals of efficacy are often unclear or inconsistent across diseases. This interactive workshop will bring together industry and clinical experts to discuss how surrogate biomarkers can be validated and implemented to enable earlier proof-of-concept studies. Attendees involved in clinical development, translational research, and biomarker discovery will benefit from practical insights into how endpoints are being evaluated.
Join this interactive workshop to:
- Understand the Phase II endpoint gap and why traditional renal outcomes are difficult to apply in early-stage trials
- Interrogate mechanistic biomarkers in cohort studies to identify potential responder patient populations and aid in Phase II trial design
- Explore how consortia such as PARASOL are validating endpoints through collaborative datasets, translational studies, and longitudinal cohorts
- Identify strategies to integrate biomarkers into trial design to generate earlier efficacy signals and support regulatory acceptance
1:30 pm Lunch & Networking
2:30 pm Workshop C: Building a Sustainable Business Model for Rare Kidney Disease Therapies
This workshop is designed for business development professionals, biotech leaders, market access teams, and clinical development strategists working to strengthen the commercial foundations of rare kidney disease therapies. While scientific innovation in nephrology is accelerating, many programs struggle to translate clinical success into sustainable reimbursement and investment due to misalignment between development strategies, payer expectations, and pricing models.
This session will explore how clinical evidence, value demonstration, and pricing strategy can be aligned earlier in development to support stronger market access and investment readiness, helping teams integrate commercial thinking across the development lifecycle.
Dive into:
- Understanding payer and HTA expectations and how these should inform evidence generation, endpoint selection, and clinical trial design
- Developing pricing and value frameworks for rare kidney disease therapies, including how to articulate clinical and economic value to payers
- Building the strategic skills to evaluate commercial viability early, helping teams align clinical development decisions with reimbursement and investment considerations