Conference Day 2

Wednesday 1st October 2025

8:00 AM Registration & Coffee

8:50 AM Chair’s Opening Remarks

  • Ron Flauto Senior Medical Director - Global Medical Sciences, Aurinia Pharmaceutical

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

9:00 AM Bridging the Gap: Rare & Genetic Kidney Disease Models for CKD Therapeutic Development

Synopsis

  • The integration of advanced molecular, genomic, and proteomic tools enhances the translatability of findings to human CKD
  • The success of preclinical models in simulating human pathologies, disease progression, and predicting outcomes is critical for the success of drug development
  • Rare genetic kidney disease models may replicate human disease biology more accurately, offering unique insights into disease mechanisms and therapeutic responses
  • These models can be leveraged to bridge the gap between preclinical findings and clinical applications

9:30 AM Demystifying Early Biomarkers in Drug Discovery

  • Rohit Gaurav Principal Scientist I, Translational Research Expert Translational Research & Data Science, Novartis AG

Synopsis

  • Role of Biomarkers in Drug Discovery
  • Limitations of Animal Models
  • Advancing Complex and Human-Relevant Models (PCKS, organ-on-chip etc.)
  • Future Directions and Key Takeaways

10:00 AM Leveraging Human Genetics and Multi-omics to Develop Precision Therapies in 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 Morning Break & Networking

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

11:00 AM Anti-suPAR Antibody to Treat Rare CKDs

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

Synopsis

  • Understanding suPAR’s role in rare glomerular diseases
  • Generation and testing of WAL0921, an anti-suPAR antibody
  • Using proteomics to understand clinical potential

11:30 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

12:00 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

12:30 PM Lunch & Networking

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

1:30 ABO-101, a novel gene editing therapy for primary hyperoxaluria type 1, is efficacious and well tolerated in NHPs and results in high fidelity editing in primary hepatocytes

Synopsis

  • Single administration of ABO-101 to NHPs results in efficient HAO1 editing, reduced GO enzyme activity, and no clinical signs or adverse events
  • mABO-101, a mouse surrogate of ABO-101, demonstrated efficient in vivo editing and reduction of urinary oxalate in juvenile and adult Agxt KO mice
  • ABO-101 is an efficient and specific HAO1 gene editor in vitro with a favorable off-target profile

2:00 PM Insights into Genetic Kidney Diseases from Whole Exome Sequencing at Scale

  • Mary E. Haas Co-Founder & Chief Medical Officer Head of Research & Development, Regeneron Genetics Center

Synopsis

  • Types of genetic analyses and their relevance for drug development
  • Utility of whole exome sequencing to identify potential drug targets and provide complementary information on mutation prevalence and penetrance

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

2:30 PM Panel Discussion and Q&A: Navigating Global Partnerships, Funding Strategies & Value Demonstration in the Rare & Genetic Kidney Disease Field
  • Jason Brown Chief Business Officer, Everest Medicines
  • Vamil Divan Managing Director - Equity Research, Biopharmaceuticals, Guggenheim Securities

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
  • 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 

3:15 PM End of Conference Day 2

3:30 PM Chair’s Closing Remarks

  • Ron Flauto Senior Medical Director - Global Medical Sciences, Aurinia Pharmaceutical
Day One
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