Pre-Conference Workshop Day

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
• Using preclinical assays to identify promising drug targets and predict their potential efficacy
• Utilizing novel preclinical models to understand the genetic heterogeneity of kidney disease to evaluate suggested therapeutic approaches
• Integrated analysis of preclinical multi-omics data from disease models to pinpoint potential targets
• Developing novel assays to evaluate the efficacy of potential single-agent therapies in vivo and ex vivo
• Exploring novel modalities that could effectively deliver therapies for increased patient comfort
• Discussing current limitations in translating identified genetic effectors into druggable targets

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

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:

• Developing platforms clinical trial recruitment, specifically in paediatric populations, to prevent full disease manifestation in adulthood
• Addressing challenges in engaging and empowering patients in their decisions to join clinical trials
• Accelerating the use of electronic medical records to reduce bottlenecks in identifying rare disease patients globally
• Working towards an integrated healthcare ecosystem where the patient community, providers, industry, and regulatory bodies collaborate effectively, with patient advocacy driving improvements in care and policy
• Lessons learnt from clinical trial outcomes that allow for better identification of patients for future trials
• Improving the standard of care for patients with rare disease and understanding best practices for effective communication to streamline care
• Harnessing AI and automation in patient identification and outcome tracking to trigger actions, alert clinical teams and suggest future actions for patients