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 Moving Beyond Diagnosis Based on Histopathology: Assessing Molecular Characteristics to Reclassify Disease Subtypes
Synopsis
- Performing biomarker studies to assess response to treatment
- Identifying and selecting patients based on biomarkers
- Molecular characteristics replacing or complementing histological diagnosis
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
11:30 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 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
4:30 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
5:00 pm Chair’s Closing Remarks
5:10 pm Scientific Poster Session
Synopsis
After the formal presentations have finished, the learning and networking carries on. The Poster Session allows you to connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships. During this session scientific posters will be presented on the very latest advancements in
drug development for rare and inherited renal disorders with a focus on better understanding and overcoming translational challenges to inform the direction of drug development for rare and genetic kidney diseases.