Explore the Agenda
Pre-Conference Day
Monday, March 16
Day One
Tuesday, March 17
Day Two
Wednesday, March 18
7:00 am Morning Check-In
7:55 am Chair’s Opening Remarks
Pioneering New Endpoints and Trial Pathways to Bring Rare Kidney Disease Therapies to Patients Faster
8:00 am Clinical Pathway to Registration: First Treatment for C3 Glomerulopathy (C3G) & Primary IC-MPGN in Patients 12+
- Achieving regulatory approval by demonstrating efficacy across all three key disease markers: 68% reduction in proteinuria, kidney function stabilization, and substantial C3 deposit clearance.
- Establishing a broad label covering adults and adolescents with C3G or primary ICMPGN, including post-transplant C3G recurrence
- Building a well-characterized safety profile supported by >2,200 patient-years of experience in approved indications
- Providing a model for rare kidney disease development, addressing conditions with high risk of progression to kidney failure
8:30 am Endpoints in Kidney Transplantation: Integrating Biopsy, Biomarkers & Surrogate Tools in Trials
- Combining kidney graft biopsy histology, de novo donor-specific antibodies (dnDSA), eGFR and proteinuria in a single composite biomarker panel for earlier prediction of long-term graft loss
- Positioning iBox, a composite biomarker panel, as a “reasonably likely surrogate endpoint” under FDA’s accelerated approval for immunosuppressive therapies in transplant settings
- Addressing technical and clinical challenges: assay standardization (dnDSA MFI), biopsy variability (Banff classification), and timing of assessment (first year after transplant)
- Evaluating how iBox can accelerate trials of therapies for antibody-mediated rejection and other transplant complications
9:00 am Imaging Kidney Fat & Function: Biomarkers for Understanding CKD
- MRI-based measures of renal sinus and perirenal fat can be easily integrated into clinical trials
- Imaging-based kidney biomarkers can provide additional mechanistic insights into disease pathophysiology, drug mode of action, and treatment effects
- Imaging biomarkers can complement, not replace, existing established biomarkers to deepen understanding and progress development
9:30 am Strategies for the Development & Evaluation of Treatments for Children with Renal Diseases
- Review ethical and legal obligations in studying drugs in children
- Discuss unique considerations for designing paediatric drug development programs
- Present innovative tools and strategies to increase the feasibility and success of pediatric drug development programs
10:00 am Morning Break & Refreshments
Track Chair:
Driving Precision CKD Research Through Biomarker Innovation and Mechanistic Profiling
10:30 am Panel Discussion: How Can Novel Modalities be Used to Meet the Unmet Needs in CKD?
- Balancing innovation with clinical feasibility and scalable development in kidney disease
- Addressing AAV’s narrow tropism: how do we translate promise into real-world therapies?
- Exploring whether targeted delivery strategies must be disease-specific to maximize impact
- Ensuring RNAi and oligonucleotide therapies achieve durable, clinically meaningful kidney knockdown
11:00 am A Patient-Centric approach to CKD Drug Discovery: Integrating Clinical Insights with Human Model Systems
- A patient-centric discovery framework to bridge the translational gap in CKD research
- Leveraging multi-OMICS analyses from large patient cohorts to understand disease mechanisms and identify novel therapeutic targets with high clinical relevance
- Utilizing advanced human model systems to recapitulate CKD molecular states
- An example application of targeting pathogenic mechanisms in the diseased kidney using in vivo systems and tailored in vitro assays
11:30 am Panel Discussion: How Can we Best Apply AI for Streamlined Drug Discovery in CKD?
- Explore how AI and machine learning are being appliedto target identification, protein engineering, and biomarkerdiscovery in CKD, with a focus on DKD
- Examine case studies and strategies from different labs and companies to understand the practical implementation andevolving performance of AI tools
- Discuss the integration of AI across preclinical and clinical stages to accelerate precision nephrology research and development
- Consider collaborative approaches and future frameworks, including shared strategies or white papers, to advance AI use in kidney disease drug discovery and beyond
Track Chair:
Harnessing Real-World & Biological Data to Redefine Evidence Generation in Nephrology Drug Development
10:30 am CKD Trials Across the Age Spectrum: Challenges & Opportunities for Drug Development
- Designing the first global chronic kidney disease trial in children using Jardiance
- Evaluating which clinical trial endpoints are clinically meaningful and acceptable to regulatory authorities for pediatric indications and labeling
- Exploring surrogate biomarkers and adaptive endpoints to reliably gauge efficacy and safety when disease progression is slow
- Maximizing real world data, trial design and drug development programs for future therapies in kidney disease
11:00 am Development of Balcinrenone/Dapagliflozin Combination in Chronic Kidney Disease
- Pre-clinical efficacy of balcinrenone, a novel MRA with a differentiated mode of action
- In phase 2 trials, balcinrenone/dapagliflozin was safe and reduced albuminuria in patients with CKD, with minor impacts on serum potassium levels
- Phase 3 development in heart failure and CKD
11:30 am PS-002: Pioneering Gene Therapy to Transform IgA Nephropathy Treatment
- Update on progress of clinical development of gene therapies for glomerular disease
- Patient perceptions of gene therapy
- Development path for gene therapies in glomerular disease
12:00 pm Lunch & Networking
BUILDING THE FUTURE OF CKD THERAPY THROUGH MECHANISM‑DRIVEN DELIVERY PLATFORMS, HUMAN‑GENETIC PATHWAYS, & EARLY INDICATORS OF TREATMENT RESPONSE
1:00 pm Precision siRNA Delivery to the Proximal Tubule via Endogenous Receptor Targeting
- Leverage endogenous receptor-mediated uptake (via megalin) to deliver ligand-siRNA conjugates directly into proximal tubule cells for high kidney exposure and gene silencing
- Achieve durable target gene knockdown (50–70%) in the kidney lasting up to four weeks from a single dose— demonstrating effectiveness of the receptor-mediated delivery strategy
- Overcome a longstanding delivery challenge by increasing siRNA accumulation in the kidney 5–30-fold compared to naked siRNA, enabling new therapeutic targets to be addressed
1:30 pm Leveraging Human Genetics to Advance Precision Medicine in CKD: Translating APOL1 & Amino Acid Transporter Insights into Clinical Progress
- Defining distinct CKD-susceptible populations through APOL1 genetic variants and exploring the potential to modulate this biology with a small-molecule inhibitor supported by favorable Phase 1 safety and pharmacokinetic results
- Interrogating the genetic and biological role of the amino acid transporter BoAT1 (SLC6A19) in CKD progression to uncover new opportunities for therapeutic intervention beyond current standard-of-care approaches
- Presenting Phase 1 clinical data and mechanistic hypotheses linking target modulation to improved kidney outcomes
- Leveraging human genetics and biomarker datasets to validate target inhibition, confirm efficacy signals, and enable precision-driven clinical development in CKD populations
2:00 pm Roundtable Discussion: Refining Biomarkers for Earlier Indication of Treatment Response to Predict Phase 2/3 Success
- Examine the limitations of current biomarkers like eGFR and albuminuria in predicting late-stage trial outcomes.
- Explore novel functional and molecular biomarkers that could better indicate clinical success in phase three.
- Address the challenges of long and large CKD trials by integrating predictive biomarkers to shorten timelines and refine patient selection.
- Discuss strategies to align biomarker discovery with emerging targets to increase translational confidence and probability of success
DRIVING PATIENT-CENTERED KIDNEY INNOVATION BY BRIDGING POLICY, SCIENCE, & COMMERCIAL STRATEGY
1:00 pm Navigating Kidney Healthcare Public Policy & Advocacy as it Pertains to Patients with Kidney Disease
- Expanding home dialysis access by addressing regulatory, reimbursement, and infrastructure barriers
- Implementing system-level strategies to reduce inequities and improve quality across kidney care delivery
- Shaping payment reform and value-based models to better align incentives with patient-centered outcomes
- Elevating kidney health in national policy agendas to ensure sustained legislative and regulatory prioritization
1:30 pm Accelerating Kidney Research Through the NKF KidneyCARE™ Study: A Platform for Patient Insights, Trial Readiness, and Real-World Evidence
- Building a national, patient-centered registry that integrates real-world data with patients’ lived experience to drive innovation across the spectrum of kidney diseases
- Enabling targeted trial awareness and recruitment through an opt-in patient community spanning all types and stages of CKD, including dialysis and transplant
- Utilizing tailored, rapid-turnaround “Pulse” surveys to capture timely patient insights on topics relevant to research and development priorities
- Creating a platform for sponsors and investigators to accelerate discovery and therapeutic development in both common and rare kidney diseases
2:00 pm Advancing Cell Therapy in CKD: Integrating Mechanistic Insights and Clinical Evidence from Phase 2 and 3 Trials
- Exploring the role of autologous cell therapy in treating advanced CKD, emphasizing its potential to preserve kidney function and delay the need for dialysis
- Presenting novel mechanistic studies that elucidate how specific renal cell populations in ProKidney’s REACT® therapy may stabilize and improve kidney function, potentially halting the progression of CKD
- Reviewing Phase 2 clinical trial results, including the REGEN-007 study, which demonstrated a statistically significant and clinically meaningful reduction in the annual decline of estimated glomerular filtration rate (eGFR) in patients with type 2 diabetes and Stage 3b/4 CKD
- Discussing the strategic design of the Phase 3 PROACT 1 trial, incorporating learnings from Phase 2 to optimize patient outcomes and streamline the clinical development pathway
2:30 pm Afternoon Break & Refreshments
Reclassifying Kidney Disease Beyond Proteinuria to Drive Targeted Therapies & Better Outcomes
3:00 pm Who Are the High-Risk CKD Patients Now? Redefining Risk Beyond Proteinuria
- Understanding what is the unmet need on top of standard of care and treatment landscape? Combinations & Polypharmacy?
- Are traditional definitions of high risk (low eGFR, proteinuria) still sufficient in the era of multi-omics and cardiometabolic overlap?
- What biomarkers and surrogate endpoints are emerging to identify and serve nonproteinuric CKD patients?
- How can we define clinical endophenotypes, such as patients with reduced ejection fraction, diabetes, or obesity—that may signal higher risk or non-response?
- What data are missing from current longitudinal cohorts (e.g., cardiovascular outcomes, repeat biopsies), and how do we fill these gaps to better guide drug development?
3:30 pm Closing the Innovation Loop: Mechanistic Trials as a Tool for Precision Medicine in 2026
- Integrating deep multiscalar data sets from biopsy cohort studies (NEPTUNE, KPMP, BEAT-DKD) has allowed to build a molecular map of the kidney in health and disease
- Mechanistic trials with entry and exit biomarkers and biopsies now allow to define treatment responses to experimental therapies
- First success in rare and common diseases will be presented
4:00 pm Panel Discussion: Data Sources in Nephrology Drug Development: Choosing Wisely, Mitigating Bias & Maximizing Impact
- Sharing experience using different data sources (clinical trials, registries, EHRs, biobanks, biopsies) for drug development from discovery through regulatory approval and post-market
- Comparative strengths and limitations of each data source: e.g. trial data offers control and randomization; registry/EHR data offers large-scale, real-world generalizability but more noise; biopsy samples offer deep mechanistic insight but are invasive and limited in availability
- Strategies to handle missing, incomplete, or biased data: statistical techniques, data linkage, standard operating procedures for sample collection, dealing with confounders, ensuring representative sampling
- How to align data sourcing strategy with regulatory and payer requirements: the evidentiary standards, reporting, validation, transparency, reproducibility
- How to gain buy-in from diverse stakeholders (industry, federal, academic centers) for data sharing and collaboration, potentially identifying a convener or honest broker
- Discussing the extent to which professional medical societies (like ASN) and initiatives (like KHI) should be involved in data generation and evaluation
4:40 pm Leveraging Public-Private Partnerships to Accelerate Kidney Disease Innovation
- Establishing public-private partnerships to advance therapies for people with kidney diseases
- Promoting patient-centered approaches in designing and developing innovative therapies
- Facilitating the development and multisector adoption of tools, data standards, and endpoints that advance new therapies
- The role of the Kidney Health Initiative (KHI) in facilitating collaboration between stakeholders
5:10 pm A Call to Action for CKD: Connecting the Dots to Achieve a Cure for CKD
- Building a unified kidney community to advance glomerular, AKI, CKM, and broader CKD innovation across the full continuum of care — from early diagnosis to regulatory and reimbursement readiness
- Leveraging collaboration across the entire kidney community to align on practical, implementable frameworks that ensure new CKD therapies can reach patients who need them without delay
- Embedding implementation planning early in development to create and continuously strengthen the substrate of next‑generation CKD therapies
- Driving collective responsibility across clinicians, developers, regulators, and advocates to ensure equitable access and sustained adoption of innovative CKD treatments