Explore the Agenda
Focus Day
Rare & Genetics Focus Day
8:30 am Check In, Coffee & Light Breakfast
9:30 am Targeting the Central cAMP Signaling Defect in ADPKD to Advance a Genotype-Agnostic Small-Molecule Therapy into the Clinic
- Directly targeting abnormally elevated cAMP through Mironid’s first-in-class LoAc small molecules to intervene in a central driver of cyst formation and growth
- Building on preclinical evidence supporting reductions in cyst formation and kidney enlargement to translate PDE4- mediated cAMP modulation into a clinical, orally available treatment strategy for ADPKD
- Advancing a genotype-agnostic approach designed for patients across PKD1, PKD2 and other genetic backgrounds to broaden therapeutic reach and complement increasingly mutation-specific genetic and RNA-based approaches emerging across the ADPKD pipeline
10:00 am Accelerating PKD Drug Development: Lessons from Clinical Trials, Biomarkers, & Regulatory Innovation in Rare Kidney Diseases
- Which innovations in rare kidney disease trial design are genuinely transferable to ADPKD? Focus on enrichment, trial duration, sample size, and innovative designs
- What would it take to establish a new surrogate endpoint in ADPKD? Compare the regulatory experience with proteinuria in IgAN and other glomerular diseases with the experience using TKV and eGFR in ADPKD
- Can regulatory precedents from other rare kidney diseases accelerate PKD drug development? Discuss accelerated approval, natural history evidence, patient-centered outcomes, and regulatory engagement
10:30 am Morning Break
11:00 am Advancing RNA, Gene Editing & Targeted Genetic Therapies in PKD to Address Disease at its Source & Deliver Durable Benefit
- Comparing mutation-specific, RNA and gene-based approaches emerging across PKD
- Overcoming delivery challenges to reach the relevant kidney cell populations safely
- Designing development strategies that can accommodate increasingly narrow genetic subgroups
11:30 am Roundtable Discussion: Matching PKD Therapies to Genetic Drivers to Advance Precision Treatment & Reach Mutation-Defined Patient Populations
- Characterizing pathogenic mutations and variants of uncertain significance to improve patient selection
- Expanding genetic testing to create trial-ready populations for mutation-specific therapeutics
- Connecting genotype to therapeutic response to support increasingly personalized development programs
12:30 pm Lunch & Networking Break
1:30 pm Transferring Lessons from IgAN, FSGS & Membranous Nephropathy to Build Faster Approval Pathways Across Rare Kidney Disease
- Comparing where proteinuria, eGFR and disease-specific biomarkers have successfully supported approval
- Examining why surrogate strategies cannot automatically be transferred between kidney diseases
- Applying lessons from successful rare renal programs to PKD, Alport syndrome and less-developed indications
2:00 pm Looking Beyond Today’s Rare Kidney Successes to Identify the Next High-Value Indications & Expand Therapeutic Innovation
- Prioritizing Alport syndrome, FSGS, membranous nephropathy, congenital kidney disease and other underserved populations
- Translating successful mechanisms from crowded indications into diseases sharing relevant biology
- Defining where industry should focus next as IgAN becomes increasingly competitive
2:30 pm End of Seminar Day
Workshop Day
Preclinical Translation, Clinical Trial Design & Novel Endpoints Workshop Day
Tailor-made for discovery scientists, translational researchers, clinical development leaders, biomarker experts, regulatory professionals and medical teams working across CKD, this year’s workshops provide a practical forum to tackle the decisions shaping renal drug development. Compare preclinical and human-relevant models, redesign trials around an evolving standard of care, and evaluate the next generation of biomarkers and endpoints to improve translation, strengthen evidence generation and accelerate more efficient, disease-appropriate clinical development.
8:00 am Check In, Coffee & Light Breakfast
9:00 am Workshop A | Benchmarking Animal, Organoid & Human-Relevant CKD Models to Improve Translation, De-Risk Target Selection & Enter the Clinic with Greater Confidence
No single CKD model captures the heterogeneity of human kidney disease. This workshop will critically compare 5/6 nephrectomy, dietary and disease-specific animal models alongside organoids, vascularised organoids, organ-on-chip systems and patient-derived approaches. Participants will explore which models answer which biological questions, where misleading translation occurs and how clinical data can be back-translated to build stronger preclinical packages before substantial development capital is committed.
What attendees will gain: A clearer decision framework for selecting and combining models according to disease biology, modality and translational objective.
Key questions:
- Which animal models best predict human CKD biology – and where does each fail?
- When should developers use disease-specific versus general CKD models?
- How mature are organoids, vascularised organoids and kidney-on-chip systems as translational tools?
- What evidence is required before non-animal models can meaningfully influence development or regulatory decisions?
- How can human clinical observations be back-translated to continuously improve preclinical models?
11:00 am Morning Break & Refreshments
11:30 am Workshop B | Redesigning CKD Trials Around an Evolving Standard of Care to Protect Trial Integrity, Reduce Patient Burden & Accelerate Development
With SGLT2 inhibitors, GLP-1s, MRAs and disease-specific therapies increasingly shaping background treatment, CKD developers must design studies that remain relevant several years after initiation. This interactive workshop will examine evolving comparator arms, placebo ethics, background therapy requirements, recruitment challenges and alternative platform, basket and adaptive approaches. Attendees will work through how smaller, better-defined populations can be studied without sacrificing statistical power or regulatory robustness.
What attendees will gain: A practical framework for designing feasible, ethical CKD trials as therapeutic choice and standard of care continue to evolve.
Key questions:
- How should background therapy be defined when standard of care could change materially during a 2–4-year trial?
- When does placebo control become scientifically or ethically inappropriate?
- Could platform, basket or adaptive trials reduce control populations while maintaining evidentiary quality?
- How can natural history or external control data support increasingly small patient populations?
- How should industry, regulators, patients and investigators align before initiating the next generation of CKD trials?
1:30 pm Lunch & Networking Break
2:30 pm Workshop C | Building the Next Generation of CKD Endpoints to Shorten Development, Expand Beyond Proteinuria & Create Regulatory-Ready Evidence
Proteinuria, UACR and eGFR have transformed kidney drug development, but they do not adequately serve every disease, mechanism or patient population. This collaborative workshop will examine GFR slope, novel tubular and molecular biomarkers, hierarchical composite endpoints, natural history datasets, real-world evidence and disease-specific surrogate strategies. Participants will explore what is needed to move promising biomarkers from biological association to accepted development tools and ultimately regulatory endpoints.
What attendees will gain: Greater clarity on selecting and validating endpoints capable of making CKD trials faster, smaller and more disease appropriate.
Key questions:
- What should replace or complement proteinuria in nonproteinuric CKD?
- When can GFR slope support shorter and smaller registrational programs?
- Which emerging biomarkers are sufficiently mature to influence Phase I/II decisions today?
- How should developers demonstrate that a biomarker meaningfully predicts long-term clinical benefit?
- Where can FDA and EMA expectations be aligned to avoid duplicative development?