Conference Day 1
Tuesday 30th September 2025
7:30 AM Registration & Coffee
8:20 AM Chair’s Opening Remarks
Highlighting the Importance of the Patient Voice in Rare & Genetic Kidney Disease to Improve Patient Engagement & Clinical Trail Enrolment
8:30 AM Making Clinical Trials a Standard of Care in Paediatric Rare Kidney Disease to Empower Patients & Improve Enrolment
Synopsis
- Discussing successful strategies to democratize clinical trial awareness such as navigation programs and user-friendly databases that centralize trial information for clinicians and patients
- Leveraging lessons learnt from the oncology field to make trials a routine part of patients care and foster a culture where trial consideration is standard in the rare kidney disease community
- Focussing on innovative solutions to enhance clinical trial matching such as centralized platforms, longitudinal databases and the emerging ‘hub and spoke’ model to improve access and accelerate recruitment
9:00 AM Panel Discussion: Ensuring the Patient Voice is Heard to Work Towards Successful Collaboration Between Industry, Academia, Clinicians and Patients
Synopsis
- Elevating the lived experience of patients to drive relevant research and diagnosis
- Developing strategies to prioritize patient preferences in trial design, care pathways and policy advocacy Outlining the role that patient advocacy can have in articulating the needs and priorities of the rare and genetic kidney
Reaffirming Past, Present & Future Challenges in a Changing Regulatory Landscape to Promote Alignment in the Kidney Disease Community
9:30 AM Clinical Trial Considerations in Cystinuria to Support Regulatory Approvals in the United States Vs Europe
Synopsis
- Highlighting differences between the US and Europe in conducting clinical trials in rare kidney diseases
- A case study focused on Cystinuria and dRTA
- Discussing the regulatory frameworks governing development and implementation for therapeutics in rare kidney disease
10:00 AM GFR Assessment at the Point of Care: Future Transdermal GFR (tGFR) Application in the Clinic
Synopsis
- Overview of the Transdermal GFR System (TGFR) including review of the transdermal GFR (tGFR) methodology, indication for use, and patient care path
- Retrospective analysis of mGFR (relmapirazin) and tGFR data as it compares to the Standard of Care in current clinical practice
- Focused clinical use cases including a description of the unmet medical need and anticipated TGFR study design in specific patient sub-populations
- Future TGFR developments
10:30 AM Speed Networking
Synopsis
Put a face to a name – this session is the perfect opportunity to get face-to-face time with key opinion leaders, leading companies, and innovative researchers in the rare and genetic nephrology field. Establish meaningful connections to build upon for the rest of the conference and gain individual insight beyond the papers and press releases into the pioneering research and technique applications.
11:00 AM Morning Break & Networking
11:30 AM Proteinuria: A Key Endpoint in Rare Glomerular Diseases
Synopsis
In recent years, proteinuria has become regulatory approvable endpoint for new therapeutics for rare glomerular diseases and considerations for drug developers include:
- Considerations of measuring urinary albumin vs. total protein
- Timed 24-hour proteinuria or albuminuria excretion vs. spot urinary protein-to-creatine ratio
- Implications of varying recommendations in KDIGO and other nephrology practice guidelines
12:00 PM Panel Discussion: Defining Surrogate Endpoints for Rare & Genetic Kidney Disease
Synopsis
- Current regulatory perspectives on surrogate endpoints such as proteinuria in rare kidney diseases
- Industry perspectives on balancing the need for a 'registrable' surrogate endpoint with the goal of achieving a 'clinically compelling' outcome for patients
- Patient views on the use of surrogate endpoints in clinical trials
12:45 PM Genetic Insights from RenasightIQ™: Etiologies and Therapeutic Implications or CKD Patients with Diabetes and Hypertension
Synopsis
Discover new real-world evidence from Natera’s RenasightIQ™, a clinicogenomic dataset linking genetic testing results with longitudinal EHR and claims data from tens of thousands of CKD patients. Two groundbreaking retrospective studies will be presented:
- Hypertensive CKD and APOL1: Data reveal a significant reduction in progression to ESKD among APOL1 dual-risk variant (DRV) carriers treated with SGLT2 inhibitors— highlighting the opportunity for biomarker-driven therapeutic positioning and trial design in high-risk populations
- Diabetes-Associated CKD and Genetic Risk: A separate analysis underscores how APOL1 and MODY variants influence CKD trajectory and dialysis risk, and how GLP-1 agonists offer significant renal protection in these genetically defined subgroups
1:15 PM Lunch & Networking
What’s Next in the Renal Renaissance? Exploring the Latest Clinical Developments to Stay Ahead of the Curve
2:15 PM Delving into Clinical Trial Design for APOL1-Mediated Kidney Disease
Synopsis
- Updates on the progression of the AZD2373 for the treatment of APOL1-Mediated kidney disease
- Delving into successful phase 2 study design in rare renal diseases
2:45 PM Navigating the Key Complexities in Clinical Trials for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Synopsis
- Explore the spectrum of ADPKD’s renal and extra-renal complications, alongside the unpredictable nature of disease progression, which complicates diagnosis and patient management
- Address the practical challenges in optimizing current treatments like tolvaptan, focusing on adherence, managing side effects, and patient selection for long-term benefit
- Beyond Kidney Function: Discuss the significant impact of ADPKD on patient quality of life, including pain and psychosocial burdens, underscoring the critical need for integrated, multidisciplinary care
3:15 PM Beyond Traditional Endpoints: MRI in Rare & Genetic Kidney Disease Drug Development
Synopsis
- The Power and Potential of Imaging in Kidney Disease Trials: Discover how advanced imaging can reveal subtle disease progression, measure treatment effects earlier than traditional endpoints, and deliver whole‑organ insights that complement biomarkers such as eGFR and proteinuria
- Current usage of kidney imaging in clinical trials: See how imaging is already shaping trials in ADPKD, CKD, and DKD by enabling precise, reproducible measurements of kidney volume, perfusion, and other structural and functional parameters across global sites
- Expanding Horizons: From Rare Kidney Diseases to Multi‑Organ Insights: Explore the potential of imaging endpoints in rare and genetic kidney diseases, and how integrating kidney imaging with assessments of metabolically linked organs like the heart and liver supports more holistic drug development strategies
3:45 PM Afternoon Refreshments
4:15 PM Development of EVER001, a Covalent Reversible Bruton’s Tyrosine Kinase (BTK) Inhibitor, for the Treatment of Primary Membranous Nephropathy and Other Rare Kidney Diseases
Synopsis
- Outlining the role of BTK in modulating B-cell pathways that are involved in the pathogenesis of autoimmune kidney diseases
- Reviewing the current clinical data demonstrating the activity of EVER001 in primary membranous nephropathy
- Looking towards expanded applications in additional rare, immune-mediated kidney disorders
4:45 PM Shifting Toward Treating Kidney Diseases Rather than Treating the Consequences of Kidney Diseases
Synopsis
- The new molecules being developed with kidney indications have shifted focus.
- Surrogate endpoints such as eGFR change, eGFR slope and proteinuria are innovating the treatment of kidney disease
- Voclosporin is a “real life” example of the use of surrogate endpoints and has changed the standard when treating lupus nephritis
- The impact of proteinuria reduction should not be underestimated, including to levels below what is recommended by guidelines