Advancing the Next Generation of Precision Kidney Disease Therapies from Discovery to Patients
The 6th Rare & Genetic Kidney Disease Drug Development Summit is the only industry-focused forum dedicated to advancing therapies for rare and inherited renal diseases. This meeting brings together leading experts working across target discovery, translational science, clinical development, regulatory strategy, and patient engagement. This includes decoding the underlying mechanistic renal biology, identifying critical surrogate endpoints in clinical trials, and addressing the practical challenges of trial design, patient recruitment, and real-world data integration to improve clinical outcomes.
With insights from 20+ expert speakers from Novartis, Takeda, Boehringer Ingelheim, Vertex Pharmaceuticals, and more, this summit offers a unique opportunity to hear how leading organizations are navigating the realities of developing treatments for rare kidney diseases. Through candid discussions on key decision points and lessons learned, attendees can benchmark their strategies and gain perspective on the approaches shaping the next wave of renal therapeutics.
Unmissable Event Highlights
Rethinking Trial Design to Overcome Rare Kidney Disease Challenges
As rare kidney disease trials face increasing pressure from small patient populations and complex study requirements, innovative approaches are essential. Drawing on experiences from organizations such as Travere Therapeutics, Vera Therapeutics, and patient-focused perspectives from KidneyFuture, this session explores how real-world data, external control arms, and adaptive platform designs are helping reduce recruitment burden, improve feasibility, and accelerate decision-making across clinical programs.
Advancing Endpoint Validation to Unlock Faster Clinical Proof-of-Concept
Validating clinically meaningful and reliable endpoints remains critical to accelerating clinical success in rare kidney disease. In this session, experts from Alexion Pharmaceuticals, Bayer, and the University of Michigan will discuss how proteinuria, tissue-based profiling, and advanced imaging approaches are being applied to support earlier proof-of-concept studies, strengthen regulatory alignment, and improve trial efficiency across renal indications.
Building Collaborative Ecosystems to Accelerate Innovation and Trial Success
As development strategies become more complex, collaboration across academia, biopharma, and clinical networks is increasingly important to therapeutic success. Featuring perspectives from Boehringer Ingelheim, NephroDI, and the University of Michigan, this session examines how cross-sector partnerships are aligning on trial design, data sharing, and the development of scalable research platforms to improve translational success and clinical feasibility in rare kidney disease programs.
Embedding the Patient Voice to Improve Trial Design and Adoption
Incorporating the patient perspective is essential to designing therapies that are both effective and feasible in real-world settings. Experts from Boehringer Ingelheim, Vertex Pharmaceuticals, Amicus Therapeutics, and Maze Therapeutics will share how patient input is shaping trial design, informing endpoint selection, and influencing treatment delivery approaches to improve adherence, experience, and longer-term outcomes.
Explore the Full Event Guide
- See how companies are tackling challenges from preclinical target validation through clinical development, payer engagement, and asset evaluation
- Preview strategies across 5+ rare kidney disease indications
- Insights from the companies and experts advancing renal pipelines
- Learn how teams are addressing surrogate endpoints, clinical trial design, and patient recruitment
- Share key insights with internal stakeholders to align your team
Attending Companies Include
Explore the Agenda
Hear the latest breakthroughs in biomarker validation, surrogate endpoints, and clinical trial design through a packed agenda featuring, 3 expert panel discussions, 3 pre-conference workshops, 2 interactive roundtables, and 12+ data-driven case study presentations.
Partner With Us
Position yourself alongside leading solution providers supporting rare and genetic kidney disease pipelines. Whether your expertise lies in preclinical or clinical research, novel biomarkers for diagnosis, stratification or monitoring, imaging, or genetic testing, ensure your brand is at the center of key conversations around clinical development, endpoint validation, and trial execution
Join Biopharma Experts
Engage with a uniquely focused audience of rare and genetic kidney disease decision-makers and be part of high-value conversations with like-minded attendees across all stages of drug development, building critical connections through dedicated networking opportunities