Explore the Agenda
7:30 am Check In, Coffee & Light Breakfast
8:20 am Opening Remarks
Validating Biomarkers, Surrogate Endpoints & Imaging Measures to Enable Earlier Proof-of- Concept for Therapeutic Efficacy
8:30 am Shortening Rare Kidney Disease Trial Timelines by Integrating Imaging & Molecular Biomarkers into Endpoint Frameworks to Generate Earlier Proof-of-Concept Data
- Evaluating Total Kidney Volume (TKV) as the current imaging-based surrogate endpoint for disease progression in ADPKD clinical trials
- Understanding how imaging endpoints correlate with renal function decline and long-term patient outcomes, supporting their role in accelerating clinical development
- Exploring opportunities to combine imaging with molecular biomarkers, including polycystin and emerging biomarkers, to improve endpoint sensitivity
- Addressing the technical and feasibility challenges of complex biomarker assays, such as exosome-based biomarkers, when implementing them in multicenter clinical trials
9:00 am Transdermal GFR Assessment at the Point of Care
- Overview of the TGFR™ System including review of the 2nd generation transdermal GFR methodology, indication for use, and patient care path
- Retrospective analysis of measured GFR (relmapirazin) and transdermal GFR data as it compares to current clinical practice
- Clinical use cases including a description of the unmet medical need and anticipated TGFR study design in specific patient sub-populations
- Future TGFR™ Developments
9:30 am Kidney Biopsy Profiling: Revealing Drug Mechanisms & Responsive Patient Subgroups in Kidney Disease Trials
- Profiling patient kidney tissue before and during clinical trials to understand molecular changes and identify early signals of therapeutic response
- Comparing trial biopsy data with longitudinal cohort datasets to determine where drugs succeed or fail mechanistically
- Advancing precision medicine by linking molecular tissue changes with clinical outcomes and guiding future biomarker-driven trial design
10:00 am Speed Networking
Put faces to names in a fast‑paced session designed to connect you with key opinion leaders, innovative researchers, and leading companies across rare and genetic nephrology. Build meaningful relationships that extend throughout the conference and gain practical insight beyond published data into emerging research and applied techniques.
10:30 am Morning Break & Networking
Decoding Rare Kidney Disease Biology by Integrating Molecular Insights & Pathways to Identify Actionable Therapeutic Targets
11:00 am Leveraging Signaling Networks to Identify & Therapeutically Target Disease-Relevant Pathways in ADPKD
- Identifying and validating disease-driving signaling pathways across rare and genetic kidney diseases
- Evaluating strategies to therapeutically modulate dysregulated pathways, including inhibition, activation, and allosteric regulation
- Understanding how targeting different points in a signaling cascade influences disease progression and treatment outcomes
11:30 am Beyond the Biopsy: Genetics as the On-Ramp to Better FSGS Cohorts
- See a new cohorting opportunity in action. Using prior FSGS trials as the anchor, explore how genetics-plus-clinical logic opens a new approach to patient cohorting.
- Watch a tiered cohort come together in minutes. Inside the kidney-native RenasightIQ™ environment, see how three tiers (genetic driver, clinical FSGS, and both) resolve a broad histologic label into higher-value subgroups.
- Leave ready to do it yourself. Learn how self-serve access lets your team define and size glomerular cohorts on your own timeline
12:00 pm Lunch & Networking
Aligning on a Changing Regulatory Landscape to Improve Approval Pathways & Development Success
1:00 pm Panel Discussion: Translating Advances in Disease Biology into Precision-Based Trial Design for Rare & Genetic Kidney Diseases
- Explore how advances in disease biology and molecular characterization are enabling more precise patient segmentation across rare and genetic kidney diseases
- Discuss how diagnostic strategies, biomarker approaches, and assay validation considerations can support the identification of patients most likely to respond to targeted therapies
- Examine how scientific, industry, and regulatory perspectives can align to inform precision-based trial design, therapeutic development strategies, and future approval pathways
1:30 pm Navigating the Clinical & Regulatory Pathway to Approval in Rare Kidney Disease
- Demonstrating efficacy across multiple endpoints to support regulatory decisionmaking in rare kidney disease
- Designing clinical programs that balance feasibility with the need for robust and clinically meaningful data
- Overcoming challenges associated with small, heterogeneous patient populations in late-stage development
2:00 pm Roundtable Discussion: Integrating Genetic Testing into Clinical Guidelines & Trial Design to Enable Precision Therapies & More Targeted Clinical Trials
Practical and highly interactive breakout roundtables where attendees can crowdsource solutions and share opinions around pre-assigned topic areas.
- How should genetic testing be integrated into routine diagnosis and clinical trial enrollment?
- To what extent are clinical guidelines (e.g., KDIGO) keeping pace with advances in genetic testing, and what gaps still need to be addressed?
- How are regulators likely to evaluate genotype-defined patient populations and nmutation-specific therapies in rare kidney disease trials?
Moderator Feedback & Audience Debate
Moderators will be assigned to each roundtable to facilitate discussion and collate
the findings. Following the roundtable discussions, they will present back to the entire
delegation and open wider audience debate
2:30 pm Afternoon Refreshments & Poster Session
Immerse yourself in an informal, relaxed session designed to spark thoughtful discussion with peers. Explore a diverse range of poster presentations or share your own work showcasing advances in therapeutic development. This session offers a valuable opportunity to connect, exchange ideas, and present your research. To submit a poster, please contact info@hansonwade.com.
Designing Patient-Centered Kidney-Targeted Therapies by Engineering Effective Renal Delivery Strategies to Improve Tissue Targeting & Treatment Adoption
3:30 pm How Close Are We to a Cure? Advances in Gene & Genetic Therapies for PKD
- Understanding why PKD is a dose-dependent disease and how this reshapes therapeutic strategy
- Differentiating emerging genetic therapies from traditional gene therapy approaches
- Exploring patient-centric delivery strategies for targeting kidney cells, including how route of administration impacts safety, tolerability, and access
4:00 pm Panel Discussion: Integrating the Patient Voice into Rare Kidney Disease Drug Development through Trial Design, Endpoint Selection, & Treatment Delivery to Improve Therapy Adoption & Satisfaction
- Incorporating patient perspectives early in drug development to ensure therapies address meaningful outcomes such as disease progression, dialysis avoidance, and quality of life
- Incorporating patient priorities and lived experience into therapy design and development strategies
- Understanding treatment burden, delivery preferences, and risk tolerance from the patient perspective