About Event

Capturing the industry’s most exciting developments to tackle rare & genetic kidney diseases

Returning for its fourth year, the Rare & Genetic Kidney Disease Drug Development Summit is leaning into the industry’s key challenges of elucidating underlying disease pathophysiology using novel tissue bioengineering approaches, genetic studies and -multi-omics to identify novel therapeutic targets.

Our 30+ expert speakers will delve into validating biomarkers of drug response and disease progression, baking endpoints and patient-centric outcome measures into clinical trial design and leveraging electronic health records for patient stratification to ultimately accelerate therapies into the clinic and towards approval!

This is your comprehensive guide to define your drug development strategy for 2025 and delve into the opportunity held by the next generation of game-changing therapeutics that will add to the arsenal of treatment options for rare renal patients. 

You'll get the chance to dive into interactive discussions on:

4th Rare & Genetic Kidney Disease Drug Development
4th Rare & Genetic Kidney Disease Drug Development
4th Rare & Genetic Kidney Disease Drug Development
4th Rare & Genetic Kidney Disease Drug Development

Investigating molecular intricacies & fundamental mechanisms of rare kidney disease in preclinical models to identify potential future therapies

Exploring genetic studies and -omic profiles to map response profiles and biomarkers of disease progression and drug efficacy

Evolving strategies for rare renal patient subgroup identification using electronic health records

Baking patient-centric outcomes and regulatory endpoints into trial design and clinical pipeline updates

New for 2024, the pre-conference workshop day will focus on topics including pinpointing molecular mechanisms underlying podocytopathies, as well as pricing, reimbursement, market access and value communication to insurers, payers and prescribers.

Returning workshops provide deep dives on industry-critical areas of evolving strategies to identify patients for trials using electronic health records, baking patient-centric outcomes into trial design, leveraging genetic and -omic patient profiles to map response profiles and recapitulating rare renal diseases using in vitro, in vivo and in silico tools.

With 30+ world-class speakers and over 80 senior attendees, join the largest community of rare and genetic kidney disease drug development experts for a deep dive into pipeline development progress, lessons learnt and novel strategies to accelerate clinical progress in orphan kidney disease populations!

Unmissable Highlights Include:

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Investigate fundamental mechanisms of disease in preclinical models to identify future therapies with Boston University, Brigham & Women’s Hospital & REVITALE Pharma

Explore genetic studies and -omic profiles to map response profiles and biomarkers with the University of Michigan & Harvard Medical School

Evolve strategies
for patient
identification
using electronic
health records
with Novartis,
Nationwide
Children’s
Hospital & Takeda

Apply patientcentric outcome measures and explore the clinical drug development landscape with Otsuka, Purespring, Maze & XORTX

Discuss market access considerations, pricing, reimbursement and value demonstration with Cypress BioPharma, SmithSolve LLC & Advicenne S.A.