Conference Day Two
Breakfast Briefing – Understanding Physicians & Primary Care Givers Perspectives
Synopsis
The Breakfast Briefings at the CKD summit is designed to foster open and insightful conversations in an intimate setting, providing a unique opportunity for attendees to interact with top physicians, researchers, primary care givers and patient advocates. In these roundtable discussions, experts will share their personal experiences, insights, and practical knowledge on key challenges and opportunities in therapy development and patient care. Whether you’re a seasoned professional or new to the field, these sessions offer a rare chance to explore real-world applications beyond the scientific content of the main program.
8:25 am Chair’s Opening Remarks
Redefining Kidney Disease Care: From Breakthrough Discoveries to Next-Gen Clinical Trials
8:30 am Panel Discussion: Addressing the Unmet Medical Needs in CKD: Defining the Residual Risks & Pathways Forward
Synopsis
- Examine the key challenges in identifying CKD patients who continue to face unmet medical needs despite advances in therapy
- Explore how biomarkers and clinical data can highlight patients with residual risk, who remain at risk for disease progression. What is needed to establish standardized criteria for identifying these high-risk patients?
- Which pathways and modes of action that should be prioritized to address unmet needs in CKD from inflammation and fibrosis pathways to alternative MOAs that could close the gaps left by current standard-of-care therapies
- Audience Input
9:00 am GFR Assessment at the Point of Care: Future transdermal GFR (tGFR) applications in the Clinic
Synopsis
- Overview of the Transdermal GFR System (TGFR) including review of the transdermal GFR (tGFR) methodology, indication for use and patient care path
- Retrospective analysis of mGFR (relmapirazin) and tGFR data as it compares to the Standard of Care in current clinical practice
- Focused clinical use cases including a description of the unmet medical need and anticipated TGFR study design in specific patient sub-populations
- Future TGFR developments
9:30 am (Virtual) Bridging the Gap: From Biomarker Discovery to Precision Medicine Implementation in CKD
Synopsis
- Examine the wealth of biomarkers discovered for CKD and their potential to drive personalized medicine—what has been achieved, and where are we falling short in clinical implementation?
- Identify key barriers to translating biomarker discoveries into actionable tools for clinical practice, including gaps in validation, regulatory approval, and integration into nephrology workflows
- Explore strategies to close the discovery-to-implementation gap, leveraging lessons from European precision medicine initiatives to improve adoption and impact in diverse patient populations
10:00 am Embedding Imaging in CKD Drug Development
10:30 am Morning Break
Track 1: Discovery, Preclinical & Translational
Advancing Kidney Disease Research with Innovative Preclinical Models Better Predicting Human Outcomes Bridging the Translational Gap
11:00 am Implementing Proteomics & High-Content Imaging in Preclinical Models to Enhance Translatability to Human Disease
Synopsis
- Discuss the significant species-specific differences in renal physiology between commonly used rodent models and humans, including variations in renal blood flow, nephron structure, and GFR which hinder the accurate representation of human kidney diseases
- Exploring novel approached to improve the relevance of preclinical models to provide a more accurate platform for studying disease mechanisms and therapeutic responses: High Content Imaging and Proteomics
11:30 am Harnessing hPSCs & Kidney Organoids: Advancing Functional Disease Models for Drug Discovery & Regenerative Medicine
Synopsis
- Explore how differentiating human pluripotent stem cells (hPSCs) into kidney organoids allows for creating ‘kidney-ina-dish’ models that closely mimic human kidney structure and function, offering a precise platform for studying kidney diseases
- Detailed use of hPSC-derived organoids in modeling genetic mutations and disease mechanisms, screening for nephrotoxicity, and evaluating therapeutic efficacy, including regenerative therapies
- Discuss future directions for kidney organoid research, including high-throughput drug screening and applications in precision medicine. Highlight how engineered mutations in hPSC lines can drive personalized treatment approaches for kidney disease
Track 2: Clinical Innovation, Regulatory & Outcomes Track
Adapting to a Changing Landscape: Innovative Trial Designs in Renal Disease Drug Development to Better Prepare for Clinical Development in a Dynamic Market
11:00 am Roundtable Discussion: Navigating Regulatory Pathways in an Evolving Multi-Drug CKD Treatment Landscape
Synopsis
This interactive discussion provides the opportunity to share challenges and brainstorm strategies for optimizing clinical trial design in a complex multi-drug treatment landscape.
Topics Include:
- Evolving CKD Regulatory Guidelines & Endpoints
- Defining Success in a Multi-Drug Treatment Era
- Innovations in Trial Design & Regulatory Pathways
11:30 am Panel & Audience Discussion: Enhancing Diversity in CKD Clinical Trials: What is Needed, What is Working, & What is Next?
Synopsis
- Explore initiatives that have increased participation of underrepresented groups in CKD clinical trials where these efforts have yielded more inclusive trial populations
- Discuss the importance of diverse representation in trials for generating more accurate and generalizable data ensuring that treatments are effective across all demographics
- Explore new and emerging methods for ensuring diverse trial participation, such as the use of decentralized trials, remote monitoring, and partnerships with local healthcare providers
12:00 pm Lunch Break
Exploring the Next Frontier in CKD: Future Directions & Emerging Therapeutic Innovations in CKD Beyond SGLT2’s
1:00 pm Revolutionizing Kidney Disease Treatment: Judo’s STRIKE Platform for Precision RNA Delivery
Synopsis
- Deep dive into Judo’s STRIKE (Selectively Targeting RNA Into KidnEy) platform and its approach to overcoming the notoriously complex nature of genetically targeting the kidney
- Explore the approach to leverage receptor-mediated oligonucleotide delivery to the kidney with ligand-siRNA conjugates that silence several target genes
- Uncover the benefits of kidney-specific delivery mechanisms and the promises to reduce the side effects commonly associated with broad-spectrum therapies, offering patients a better quality of life
1:30 pm Emerging Targeted Approaches in Renal Diseases: Pioneering Innovations for 2025 & Beyond
Synopsis
- Delve into the latest advancements in understanding molecular mechanisms driving renal diseases, including fibrosis, inflammation, and metabolic dysfunction, to identify groundbreaking therapeutic targets
- Examine the journey from target identification to drug development, emphasizing innovative approaches like precision medicine, biologics, and combination therapies tailored to address unmet needs in renal disease populations
- Consider how emerging technologies, biomarkers, and regulatory insights are reshaping the future landscape of renal disease treatments, enabling more personalized, effective, and sustainable therapeutic strategies
2:00 pm Targeting MicroRNAs in CKD: Leveraging RNA Biology for Precision Therapeutics in Kidney Disease
Synopsis
- Explore how Regulus Therapeutics’ antisense oligonucleotide platform targets microRNAs to regulate gene expression in CKD pathways like fibrosis and inflammation, aiming to limit off-target effects and directly impact cellular mechanisms
- Discuss advancements in selective kidney targeting, where oligonucleotides preferentially bind to microRNAs in renal cells, achieving localized therapeutic action and minimizing systemic effects
- Review preclinical and clinical data on Regulus’ focus on orphan kidney diseases, including ADPKD, and highlight the potential of microRNA-targeted therapies as disease-modifying treatments in CKD
2:30 pm Panel Discussion: What’s Next for CKD Therapeutics – Predicting the Future of Innovation & Treatment
Synopsis
- Reflecting on the Current State of CKD Research and Treatment: How are the innovations we’ve explored today shaping the future of CKD treatment, and what areas are poised for the most rapid advancement in the next 5–10 years?
- Emerging Therapeutics and Combination Therapies: As new therapeutics, like GLP-1 receptor agonists and RAAS inhibitors, gain traction, what novel combinations or therapies hold the most promise for enhancing outcomes in CKD patients?
- Addressing Unmet Needs in CKD: What are the most critical unmet needs in CKD today? How can upcoming research and innovations, including precision medicine approaches and advanced biomarkers, fill these gaps?