Sorin Fedeles
Executive Director Critical Path Institute (C-Path)
Seminars
Thursday 16th October 2025
PKD Trial Simulator: Insights from PKD Consortium as Tools for CKD Drug Development
9:50 am
- Leveraging the PKD Outcomes Consortium’s joint‐model linking longitudinal total kidney volume (TKV) growth to eGFR decline or ESRD to enrich trial populations
- Using a Clinical Trial Simulator tool to predict outcomes, design shorter or smaller trials, and optimize inclusion criteria
- Qualifying TKV as a prognostic biomarker with FDA and EMA to enable regulatory efficiency and accelerated pathways
- Integrating patient registry, natural history, and trial data into unified models for trial design and drug response prediction.
Thursday 16th October 2025
Defining the PKD Dosage Threshold: Mechanistic Drivers & Therapeutic Strategies to Elevate PC1 in ADPKD
11:30 am
- Dissecting the two‑hit model and how sub‑threshold PC1 levels initiate cystogenesis
- Characterizing PKD1/PKD2 dosage requirements that modulate disease penetrance and progression
- Reviewing emerging therapeutic approaches designed to increase functional PC1 and restore cellular homeostasis
- Integrating mechanistic insights with translational efforts to accelerate dose‑targeted ADPKD therapies
