Conference Day 2

Wednesday 1st October 2025

8:00 AM Registration & Coffee

8:50 AM Chair’s Opening Remarks

Examining Preclinical & Translational Strides in Rare & Genetic Kidney Disease Drug Development to Inform Next-Generation Therapies

9:00 AM Optimizing Translatable Models of Rare Kidney Diseases to Support a Strong IND Data Package

Synopsis

  • Discussing the benefits of carrying out a multifaceted approach integrating biochemical, cell-based, mouse, microfluid chips and organoids to build a comprehensive and compelling data package
  • Examining the potential of chip systems derived from human patient cells to test drug candidates and identify suitable biomarkers
  • Understanding how the evolving nature of using chip systems can be used in conjunctions with traditional PKPD studies to support IND applications 

9:30 AM Improving the Predictive Power of Preclinical Studies through Human Cell-Based Assays in Renal Diseases

  • Rohit Gaurav Principal Scientist (Translational Research Expert), Novartis AG

Synopsis

  • Examining the limitations of animal models to predict human response to therapeutics in rare disease with complex pathophysiology
  • Exploring human cell-based assays such as kidney derived organoids and isolated glomeruli, to better demonstrate disease mechanisms in vitro for more translatable preclinical data
  • Discussing the future considerations for these assays such as standardization, validation, and regulatory opinions 

10:00 AM Harnessing Multiomics to Prioritize & Validate Potential Novel Drug Targets for Genetic Kidney Disease

  • Maarten Hoek Senior Vice President & Head Of Research, Maze Therapeutics

Synopsis

  • Advancing novel renal therapeutics through leveraging patient profiles through omics and natural history of disease studies in paediatric genetic patient populations to identity therapeutic targets
  • Using multi-omics to understand specific molecular mechanisms and affected pathways driven by rare mutations in heterogeneous kidney diseases
  • Using multi-omics to uncover biomarkers for rare disease trials for a sensitive and specific approach for disease progression tracking and target engagement assessment

10:30 AM Atacicept in IgAN: Evaluating Safety and Efficacy for High-Risk Patients with Persistent Proteinuria

  • Chris Mix Executive Vice President – Clinical Development, Vera Therapeutics

Synopsis

  • Running multinational, randomized, double-blind, placebo-controlled Phase 2b study assessing atacicept in IgAN patients
  • Focussing in on high-risk IgAN patients who continue to experience persistent proteinuria, indicating ongoing disease progression
  • Evaluating the safety profile and efficacy of atacicept in managing disease progression in this specific patient group

11:00 AM Morning Break & Networking

Delving Into Novel Modalities & Upcoming Therapies to Treat Rare & Genetic Kidney Disease with a Precision Medicine Approach

11:30 AM Anti-SuPAR Antibody to Treat FSGS, IgAN and PMN

  • Alex Duncan Associate Director Of Digital Creative Operations, Michael Kors

Synopsis

  • Understanding SuPAR’s Role in Glomerular Diseases
  • Mechanism of Action for Anti-SuPAR Antibodies
  • Clinical Potential and Therapeutic Implications

12:00 PM Advancing Precision Nephrology & Podocyte Targeted Gene Therapies

Synopsis

  • Sharing data on PS-001 for NPHS2 driven Focal and Segmental Glomerulosclerosis and PS-002 for complement driven glomerular diseases
  • Exploring Phase 1 trial design considerations and including patient input
  • Discussing next-steps in gene therapies for glomerular diseases

12:30 PM The Impact of Factor H Related Proteins in the Pathogenesis of IgA Nephritis

Synopsis

  • Pioneering Factor H targeted therapies as a vital regulator of the complement system and preventing immune overactivity by controlling protein activation and protecting healthy cells
  • Harnessing important anti-inflammatory and anti-thrombotic properties, contributing to broader physiological balance as a therapeutic method

1:00 PM Lunch & Networking

Reaffirming the Importance of Genetic Understanding in Rare Kidney Disease to Inform Target Identification & Patient Classification

2:00 PM Roundtable Discussion: Accelerating the Role & Implementation of Genetic Testing in Rare Kidney Diseases

Synopsis

  • This session provides the opportunity for smaller groups to come together, share learnings, analyze, debate, and discuss the below topics. Participants will discuss the questions provided for 15 minutes, followed by 15 minutes of feedback.

2:30 PM Panel Discussion: From Discovery to Clinical: Integrating Genetics into Nephrology to Boost Early Detection & Uncover Novel Targets

  • Mary Haas Associate Director - Cardiometabolic Metabolic and Skeletal Disease Genetics, Regeneron Pharmaceuticals
  • Maarten Hoek Senior Vice President & Head Of Research, Maze Therapeutics
  • Sean Eddy Associate Research Scientist, University of Michigan

Synopsis

  • Exploring the increasing recognition of genetic etiologies in kidney disease and the need for strong genetic testing in nephrology practice
  • Discussing how nephrologists can effectively identity patients who would benefit from genetic evaluation and understand appropriate timing and indications for testing
  • Delving into the core components of genetic counselling to improve understanding of the support that is offered in these meetings to facilitate informed referrals and collaborative care management
  • Highlighting how the integration of genetic insights can improve diagnostic accuracy
  • Using genetic testing data to inform target validation and discovery for the next generation of genetic kidney disease therapeutics 

3:00 PM Insights into Kidney Disease from Whole-Exome Sequencing at Scale

  • Mary Haas Associate Director - Cardiometabolic Metabolic and Skeletal Disease Genetics, Regeneron Pharmaceuticals

Synopsis

  • Leveraging genetic and -omic data from natural history studies across diverse rare kidney diseases to create models that predict mechanistic markers of patient response
  • Identifying correlations between patient gene profiles and biomarkers that can predict their likelihood of response to specific therapies
  • Utilizing genetic and -omic signatures to develop targeted patient selection strategies for more efficient and effective clinical trials in rare kidney diseases

Navigating the Growing International Rare & Genetic Kidney Disease Field Through a Commercial Partnerships Lense

3:30 PM Panel Discussion and Q&A: Navigating Global Partnerships, Funding Strategies & Value Demonstration in the Rare & Genetic Kidney Disease Field

Synopsis

  • Delving into challenges and best practices for safeguarding intellectual property during global collaboration efforts in the development and commercialization of therapies for renal disease
  • Addressing the reluctance in conducting head-to-head trials
  • Exploring alternative strategies for demonstrating clinical and economic value of novel drug mechanisms compared to existing treatment options
  • Discussing innovative funding models, partnership strategies and approaches to attract investment in a competitive landscape
  • Evaluating strategies for fostering partnerships and expanding clinical trials and market access in Asia 

4:15 PM Chair’s Closing Remarks

4:20 PM End of Conference Day 2

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