8:25 am Chair’s Opening Remarks

  • Manish Maski Head, Global Rare Nephrology Medical Affairs, Sanofi

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

  • Matthias Kretzler Professor of Internal Medicine & Bioinformatics, University of Michigan

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?

  • Aliza Thompson Deputy Director of the Division of Cardiology & Nephrology, FDA

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

  • Aliza Thompson Deputy Director of the Division of Cardiology & Nephrology, FDA
  • Manish Maski Head, Global Rare Nephrology Medical Affairs, Sanofi
  • Richard Philipson Chief Medical Officer, Calliditas
  • Nader Najafian Executive Director, Clinical Development Sciences, Alexion Pharmaceuticals

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

  • Rachel Lennon Professor of Nephrology, Director of the Wellcome Centre for Cell-Matrix Research, University of Manchester

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

  • Benjamin Humphreys Joseph Friedman Professor of Renal Diseases in Medicine & Chief, Division of Nephrology, Washington University
  • Rachel Lennon Professor of Nephrology, Director of the Wellcome Centre for Cell-Matrix Research, University of Manchester
  • Anna Greka Institute Member, Director of the Kidney Disease Initiative, Broad Institute of MIT & Harvard

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

  • Anna Greka Institute Member, Director of the Kidney Disease Initiative, Broad Institute of MIT & Harvard

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

  • Libby Valenti Associate Director, Clinical Strategy and Development, Natera
  • Uptal Patel Head of Early Clinical Renal, CVRM, AstraZeneca
  • David Goldstein Chief Executive Officer, Actio Biosciences

Synopsis

  • Pre-clinical applications
  • Clinical applications

4:30 pm Employing A “Kidneyomics” Approach to Nephrotic Syndrome

  • Matt Sampson Associate Professor of Pediatrics, Harvard Medical School

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

5:30 pm Chair’s Closing Remarks

  • Manish Maski Head, Global Rare Nephrology Medical Affairs, Sanofi