Explore the Agenda
7:30 am Check In, Coffee & Light Breakfast
8:20 am Opening Remarks
Enhancing Recruitment & Trial Efficiency in Rare Kidney Disease Studies by Leveraging Global Patient Networks, Real-World Data & Innovative Trial Designs to Accelerate Patient Enrollment & Evidence Generation
8:30 am Implementing Master Protocols and Bayesian Clinical Trial Designs with Goal of Accelerating Clinical Development in Rare Kidney Diseases
- Leveraging Learnings from Oncology: Applying Basket, Umbrella, and Platform Trial Designs to Renal Disease
- Implementing Bayesian Clinical Trial Designs: Interpreting Recent Regulatory Guidance and Real-World Examples
9:00 am Reducing Recruitment Burden in Rare Congenital Kidney Disease Trials by Leveraging Natural History Data & External Control Arms to Minimize Placebo Requirements & Accelerate Patient Enrollment
- Leveraging natural history datasets, longitudinal patient registries, and real-world evidence to support external control cohorts in rare congenital kidney disease trials, including CAKUT
- Addressing recruitment challenges in small, heterogeneous, and pediatric populations by reducing reliance on large placebo arms and improving feasibility through smarter trial design
- Exploring how external control strategies, patient enrichment approaches, endpoint development, and public-private partnerships can strengthen trial readiness and accelerate future studies in congenital kidney disease
9:30 am Maximizing Asset Value in Rare Kidney Disease: Expanding from IgA to New Indications & Designing Proof-of-Concept Trials to Accelerate Development
- Leveraging clinical success in one indication to de-risk expansion into new rare kidney diseases
- Designing efficient proof-of-concept trials in small, heterogeneous patient populations
- Aligning indication expansion strategies with regulatory expectations and endpoint selection
- Balancing speed, cost, and probability of success when advancing pipeline-in-aproduct strategies
10:00 am Morning Break & Networking
Rethinking Evidence Generation for Small Patient Populations to Improve Trial Success & Enable Broader Patient Access: From Pediatric Trials to Transplant Outcomes
10:30 am Clinical Trial Design Considerations for Small Populations in Rare Kidney Disease
- Building multi-stakeholder alignment to advance clinical trial design in small kidney disease populations
- Moving beyond the status quo with bold, innovative approaches for rare population trials
- Embedding patient voice and pragmatic design principles to make trials feasible and meaningful
11:00 am Roundtable Discussion: Managing Complications in a High-Risk, Low-Volume Population to Improve Long-Term Outcomes in Kidney Transplant Patients
- Exploring the clinical burden and heterogeneity of post-transplant complications, including chronic rejection, delayed graft function, and viral infections
- Examining the challenges and opportunities in developing therapies and generating evidence in small, high-risk patient groups
- Identifying how trial design, biomarkers, and real-world data can support better outcomes
Expanding Data-Sharing Infrastructure & Clinical Networks to Improve Trial Success
11:30 am Fireside Discussion: Bridging the Gap: Aligning Academia, Industry, & Clinicians to Accelerate Clinical Innovation
- Rethinking collaboration: Navigating the tension between publication, intellectual property, and competitive advantage in academia–industry partnerships
- Empowering clinicians as partners: Building a more engaged, research-active clinical community, particularly in rare diseases, genetics, and biomarker-driven care
- Creating a connected ecosystem: Developing sustainable pipelines for data sharing, translational research, and crosssector collaboration to drive real-world impact
12:00 pm Roundtable Discussion: Building Effective Public–Private Partnerships to Accelerate Biomarker Discovery & Clinical Trial Readiness in Rare Kidney Disease
- How can academia, industry, and patient groups effectively collaborate to generate and share high-quality datasets while maintaining competitive advantages?
- What are the biggest barriers to building and scaling shared research platforms (e.g., data standardization, access, governance), and how can these be overcome?
- How can stakeholders align on the validation and use of biomarkers to support clinical trial design and regulatory acceptance?
12:30 pm Lunch & Networking
Improving Predictability in Rare Kidney Disease Drug Discovery by Integrating Human Data, Advanced Disease Models & Reverse Translational Approaches
1:30 pm Designing Preclinical Studies to Robustly Inform Clinical Success in Alport Syndrome
- Leveraging highly translational models that closely mirror human Alport syndrome to enhance predictive value
- Testing therapies on top of standard of care by incorporating ACE inhibitors or ARBs to reflect real-world clinical settings
- Predefining clinically relevant endpoints by prioritizing GFR, albuminuria, and biomarkers or structural changes over retrospective data selection
- Focusing on GFR as the key outcome to enable robust longitudinal assessment and better predict clinical and regulatory success
2:00 pm Roundtable Discussion: Integrating Human Evidence & Translational Models with AI to Improve Target Identification
- Where are organizations successfully integrating AI from early discovery through to clinical validation?
- What challenges remain in applying AI to integrate translational model data with human clinical and real-world datasets
- How can AI-driven insights be validated and translated into confident decisions when advancing targets into clinical development?
2:30 pm Targeting Undruggable Targets: Advancing Kidney-Targeted Therapies Through Molecular Engineering to Overcome Tissue Targeting Barriers
- Applying molecular engineering approaches to address “undruggable” targets
- Addressing barriers to renal tissue targeting
- Targeting dysregulated/diseased cells to restore renal function
3:00 pm Afternoon Break & Networking
Aligning Investment & Market Access Strategies to De-Risk Development & Ensure Patient Access
3:30 pm Fireside Discussion: From Asset Evaluation to Integration: How Strategic Dealmaking Accelerates Rare Kidney Disease Drug Development
- Evaluating rare kidney disease assets: What factors beyond clinical stage influence portfolio fit and investment decisions
- Cross-functional diligence in action: How business development, clinical, regulatory, and safety teams collaborate to assess opportunities under tight timelines
- From deal to development: Best practices for integrating newly acquired programs and maintaining momentum in clinical trials for diseases such as IgA Nephropathy and Focal Segmental Glomerulosclerosis