8:25 am Chair’s Opening Remarks
Uncovering Early Biomarkers to Evaluate Therapeutic Target Engagement & Delineating Evolving Regulatory Pathways to Validation
8:30 am GFR, Albuminuria & Beyond: Paving the Way to Validation of Early Biomarkers of Disease Progression & Therapeutic Efficacy
Synopsis
- Where are we now? Interrogating changes in GFR and albuminuria as early biomarkers and surrogate endpoints in rare and inherited renal disorders
- Validating biomarkers in large cohort studies to deeply characterize disease etiology and progression
- Stronger together: promoting sharing of biosamples and advancing centralized biobanks
9:00 am Navigating Enrichment of Clinical Study Populations to Establish Drug Efficacy in a Precision Medicine Era
Synopsis
- Implementing prognostic enrichment design to execute trials with fast progressors
- Enrolling clinical trial populations based on biomarker profiles to facilitate clinical
trial efficiency - Reducing heterogeneity with less restrictive entry criteria to monitor safety and efficacy
9:30 am Regulatory Perspective on Endpoint Development: How is the Regulatory Landscape Evolving with Advances in Research?
Synopsis
- Establishing a clinical pathway to correlate promising biomarkers with clinical outcome
- Advancing understanding of efficacy with the endpoints we already have
- Evaluating the regulatory landscape for paediatric and ultra-rare clinical studies and driving diversity in clinical trials
10:00 am In Person Structured Networking
10:30 am Morning Break & Networking
11:00 am Panel Discussion: Navigating Earlier Endpoint Development to Predict Clinical Outcome in Shorter Clinical Trials
Synopsis
- Can we move towards harmonizing global regulatory endpoints?
- Non-invasive imaging, patient-reported outcomes, albuminuria and UACR: driving progress in elevating alternative outcome measures
- Can we incorporate patient reported outcomes in symptomatic kidney diseases?
12:00 pm Lunch & Networking
Mice are Not Men: Recapitulating Genetic Mutations in Preclinical Models to Interrogate Mechansims & Screen Targets
1:00 pm Building a Platform of Complementary Experimental Systems to Elucidate Disease Pathogenesis in Genetically Driven Disorders
Synopsis
- Identifying appropriate experimental systems for preclinical studies
- Investigating basement membrane biology and pathobiology
- Therapeutic approaches to preserving basement membrane function
1:30 pm Zebrafish & Other Model Systems as Tools for Personalized Medicine
Synopsis
- Model systems for individualizing therapeutic strategies
- Using zebrafish to diagnose individual genetic sub-types in rare disease
- Personalized organ-on-a-chip models for rare disease
2:00 pm Panel Discussion: What is the Future of Preclinical Models in Genetically Driven Renal Disorders? Elucidating Disease Mechanisms & Navigating the Translational Gap
Synopsis
- What is the best platform to mimic human physiology and tailor models with disease specific mutations?
- From genetically modified mice to pluripotent stem cell-derived organoids: evaluating current capabilities to investigate disease mechanisms and screen novel therapeutics
- Is mass production of organoids the future of predicting efficacy or an additional tool?
Clarifying the Molecular Composition of Disease Sub-Types with Proteomics & Transcriptomics to Understand Functional Impact of Genetic Variants
2:30 pm Genes to Treatments: The Arc of Discovery for Kidney Diseases
Synopsis
- What are the fundamental mechanisms that regulate the trafficking and degradation of misfolded protein cargoes?
- How can we harness next-generation technologies to develop treatments and cures for genetically-defined diseases?
3:00 pm Afternoon Break & Networking
4:00 pm Panel Discussion: Genetic & Medical Real World Data Driving Therapeutic Development
Synopsis
- Pre-clinical applications
- Clinical applications
4:30 pm Employing A “Kidneyomics” Approach to Nephrotic Syndrome
Synopsis
- Recognizing that NS has the genetic architecture of a rare, complex disease and taking advantage of that for gene mapping
- Leveraging kidney single-cell sequencing technologies to elucidate pathophysiology of the genetic drivers of NS
- Democratizing and accelerating genomic discovery for NS through multipopulation analyses and data sharing
5:00 pm APRIL: Normal Physiological Functions & its Pathological Role in IgAN
Synopsis
- Physiological role of B Cell growth factors – APRIL and BAFF
- Examining the role of APRIL in IgA Nephropathy
- Considering rationale for inhibiting APRIL in IgA Nephropathy