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 leaned 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 delved 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 was 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.
Attendees got the chance to dive into interactive discussions on:
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 focused 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 provided 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, attendees joined 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!
2024 Unmissable Highlights Included:
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.
Who Could You Have Met?
