Pre-Conference Workshop Day
Workshop A
8:00 AM Advancing Novel Therapies in Rare & Genetic Kidney Disease Through Increased Mechanistic Understanding & Novel Target Identification
Synopsis
Join this workshop to deepen your understanding of the scientific strategies and innovative approaches being used to develop the next-generation of therapies for rare and genetic kidney diseases, ultimately aiming for more effective and patient-friendly treatments.
- Diving into mechanistic insights such as in vivo and in silico modelling to understand the molecular pathways to identify key intervention points for novel therapies
- Integrated analysis of multi-omics data and data from disease models to pinpoint potential targets
- Using human data to validate promising drug targets and predict their potential efficacy
- Utilizing novel preclinical models to evaluate suggested therapeutic approaches
- Discussing current limitations in translating identified genetic effectors into druggable targets
- Patient suitability for novel clinical trials through biomarker profiling
- Implementing a mechanistic trial platform for testing novel therapies in rare kidney diseases
10:30 AM Morning Refreshments
Workshop B
11:00 AM Pioneering Predictive Biomarkers & Surrogate Endpoints in Rare & Genetic Kidney Disease for Streamlined Translational Development
Synopsis
Join this workshop to gain critical knowledge and practical strategies for leveraging predictive biomarkers and surrogate endpoints to significantly streamline the translational development pathway for therapies in rare and genetic kidney diseases, ultimately benefiting patients by accelerating the availability of new treatments.
- Defining appropriate early and reliable biomarkers to predict disease progression and response to therapies to design efficient clinical trials
- Discussing novel endpoints for renal disease trials to reduce follow-up periods, cost, and complexity in clinical trials
- Defining appropriate surrogate endpoints for clinical trials in rare and common kidney diseases
- Identifying and validating surrogate endpoints that correlate with long-term outcomes in rare diseases that lack natural history data
- Developing companion diagnostics alongside new drugs to reliably identify the specific genetic mutations or biomarkers that predict a patient’s response to a drug
- Using patient registries and designing innovative clinical trials with novel endpoints that could translate findings from the rare disease space to the broader chronic kidney disease population
1:30 PM Lunch & Networking
Workshop C
2:30 PM Overcoming Obstacles in Rare Kidney Disease Trials: From “One Patient, One Site” to a Collaborative Future
Synopsis
Join this workshop to gain actionable insights to leverage innovative approaches in patient identification and clinical outcome tracking, contributing to more efficient, integrates and patient-centered healthcare ecosystem, leading to better experience and improved outcomes for individuals living with rare renal diseases.
This workshop will gather experts to discuss:
- Exploring the economic and logistical burdens of the current clinical trial model, where a single patient may be the sole participant at a given research site
- Discussing the development of new models like "Centers of Excellence" and the "spoke and hub model" to streamline patient recruitment and trial management
- Examining how CROs can move beyond a large-scale approach and build a more intimate, site-specific model tailored to the unique challenges of rare kidney disease
- Defining how pharmaceutical companies can actively support and fund these new models to reduce recruitment time, lower costs, and identify the most suitable patient populations
- Identify actionable strategies for all stakeholders, from trial sponsors to investigators, to improve efficiency and accelerate the delivery of new therapies to patients