8:00 am Registration & Morning Coffee
8:50 am Chair’s Opening Remarks
Elevating the Patient Voice to Develop Drugs for the Rare & Genetic Kidney Disease Space Using a Human-Kind Approach
9:30 am Accelerating Clinical Trials: The Power of Data Driven Patient Recruitment
Synopsis
- Understanding the value of patient engagement through different experiences in therapeutic areas including Fabry disease, IgAN, Kidney Transplantation, APOL1, and Diabetic Kidney Disease
- Discussing the importance of creating a holistic approach to patient engagement strategies
- Developing trust in patient communities
Uncovering Fundamental Mechanisms of Disease Pathophysiology to Identify Therapeutic Target Pathways
10:00 am Applying Systems Biology Approaches to Identify Injury Mechanisms & Glomerular Extracellular Matrix (ECM) Modulation as a Potential Therapeutic Approach for Proteinuria Reduction in Idiopathic Nephrotic Syndrome
Synopsis
- Isolating glomerular tissue to enable discovery-based studies to identify potential therapeutic targets for glomerular disease
- Analyzing rat glomerular transcriptomic data to understand molecular pathway dysregulation and cell types affected
- Recognizing amelioration of dysregulated glomerular ECM dynamics as a potentially targetable pathway for proteinuria reduction in idiopathic nephrotic syndrome
10:30 am Structured Speed Networking
Synopsis
This informal session provides the perfect opportunity to connect with industry frontrunners and key opinion leaders in the rare and genetic kidney disease drug development field. Establish meaningful connections to build upon at the rest of the conference and gain exclusive first-hand insights into the latest research and developments driving progress in the space.
11:00 am Morning Coffee Break
11:30 am Inhibiting the Protease Furin to Treat Anemia of Inflammation & Block TGFBeta Activated Kidney Fibrosis
Synopsis
- Understanding how anemia of inflammation caused by hepcidin can be treated
- Discovering how the protease furin can be inhibited by repurposed drugs
- Highlighting that furin inhibition can also inhibit other causes/symptoms of CKD by inhibiting TGF-beta and matrix metallo-proteinases that cause kidney fibrosis
Pre-Clinical Updates on Next-Generation Approaches Poised to Expand Therapeutic Options for Rare & Genetic Kidney Diseases
12:00 pm Biomarkers for Proof-of-Mechanism & Proof-of-Concept Trials in IgA Nephropathy
Synopsis
- Pinpointing the criticality of treatment response biomarkers in the development of promising investigational therapeutics for IgA nephropathy
- Using biomarkers to support drug development decisions, demonstrate mechanisms of drug action and predict kidney function & kidney tissue damage
- Discover how integrating biomarker insights from clinical trials and real-world studies will accelerate the development of therapeutics and biomarkers with the potential to improve the lives of patients living with kidney diseases
12:30 pm Quantifying Podocyte Targeting by AAV in Mouse Models of Nephrotic Syndrome
Synopsis
- Quantifying podocyte tropism of AAV on a single-cell level
- Making AAV mediated gene replacement therapy a viable option for many forms of monogenic nephrotic syndrome
- Improving podocyte tropism for AAV to move the field forward
1:00 pm Lunch & Networking Break
2:00 pm Harnessing Podocytes to Deliver Gene Therapy
Synopsis
- Exploring data to date suggesting kidney targeted gene therapy is feasible
- Demonstrating that kidney targeted gene therapy can achieve long-term efficacy
- Sharing case studies of podocyte targeted gene therapies
Exploring the Clinical Landscape to Gain Insights Into Game-Changing Therapies on the Horizon
2:30 pm Targeting cAMP in ADPKD by Small Molecule Activation of Long Form PDE4 Enzymes
Synopsis
- Highlighting why cyclic AMP is important in ADPKD
- Discussing the target and how to modulate cyclic AMP by allosterically activating phosphodiesterase 4
- Sharing data around compound activity in animal and cellular models
2:30 pm Proteomic Insights Into Causality & WAL0921 Treatment Effects in suPARDriven CKD
Synopsis
- Further evidence for suPAR driven progression in rare kidney disease indications
- WAL0921 as a suPAR reducing agent in kidney therapy
- Proteomic data insights into mechanism of action of anti-suPAR treatment
3:30 pm Afternoon Networking Break & Poster Session
Synopsis
Want to share your work but not ready for the big stage just yet? The Scientific Poster Session is your prime time to share your work with peers from discovery, preclinical, translational and clinical backgrounds, all working on developing intermediate and late-stage rare and genetic kidney disease therapeutics using a diversity of approaches. Get their thoughts on how you can accelerate the progression of your drug pipelines and build connections for potential collaborations to expand your rare and genetic kidney disease R&D pipelines.
4:00 pm Zetomipzomib, A Selective Inhibitor of the Immunoproteasome: Preclinical & Clinical Activity in Lupus Nephritis
Synopsis
- Demonstrating activity of zetomipzomib in a mouse model of lupus nephritis and impact on lupus gene signature pathways
- Summarising clinical data from lupus nephritis patients in Mission study
- Providing overview of ongoing PALIZADE clinical trial: a Phase 2b placebo-controlled study in lupus nephritis patients