Pre-Conference Workshop Day

Workshop Day Track A

Tuesday 12th September 2023

8:00 am
Workshop A: Beyond One Size Fits All: Deliberating the Feasibility of Gene Editing & Therapy in Tackling Rare & Genetic Kidney Disease

  • Julie Lin Global Project Head, Rare Disease and Rare Blood Disorders, Sanofi
  • Julian Hanak Chief Executive Officer, Purespring Therapeutics
  • Florin Craciun Senior Scientist, Sanofi

Synopsis

Despite the upwelling investment in gene therapy within different disease areas, there remains a ‘lag’ with kidneys. Join this interactive deep dive to understand what’s been holding back the development of gene therapy for kidney diseases and its potential applications. 

  • Highlighting the various rare and genetic kidney diseases that could be amenable to viral or non-viral therapeutic approaches
  • The importance of gene therapy and its administration in the kidneys for monogenic inherited kidney disorders 
  • Using podocytes in the glomerulus as a cell factory to express monogenic corrective genes
  • Evaluating the methods of delivery of CRISPR to kidneys 
  • Controlling gene editing events within the genome and avoiding off-target effects 
  • How can these therapies be personalized to individual patients to develop functional and validated biomarkers

11:30 am
Workshop B: Exploring the Full Potential Multi-Omics Analyses Hold as Discovery & Analytical Tools to Advance Therapeutic Development

  • Sean Eddy Assistant Scientist - Research, University of Michigan
  • Mohit Mathur Director, Clinical Development, Visterra

Synopsis

Explore the advances in transcriptomics, genomics, and protein omics to identify novel genes associated with kidney diseases and the potential use of artificial intelligence to revolutionize the kidney disease field and to streamline drug discovery efforts.

  • Leveraging the advances in comprehensive kidney omics by analyzing large datasets to accurately understand the relationship between genotype and phenotype 
  • How can artificial intelligence be used to assist nephrologists in providing optimal personalized care for patients

3:00 pm
Workshop C: Deliberating the Pros & Cons of Increasing the Administration of Genetic Testing to the General Population to Reveal Causative Pathogenic Variants in Kidney-Related Genes

  • Manish Maski Head Of Global Medical & Rare Nephrology, Sanofi Genzyme
  • Ali Gharavi Professor - Medicine Research, Columbia University Medical Center

Synopsis

Undeterred by the decreasing costs, genetic testing still isn’t 

considered routine. Join this workshop to find out why and what’s being done to influence this.

  • Understanding the recent developments in using genetic testing to find the right patient population 
  • Could an increase in genetic testing decrease the misdiagnosis of Alport Syndrome? 
  • Exploring the additional genes and panels for enhanced diagnosis 
  • Blessing or curse: Why get tested for something that doesn’t have a cure?
  • How can genetic testing aid in clinical trials patient selection for orphan renal disease?

Workshop Day Track B

Tuesday 12th September 2023

8:00 am
Workshop D: Amplifying Patients’ Voices: Understanding Patients’ Perspective & Experience to Optimise Treatment

Synopsis

Improving treatment by strengthening the relationship between patients, nephrologists, and drug developers. Attend this workshop to hear directly from patients with rare and genetic kidney disease to better shape the future of renal treatment.

  • Engaging patients in every step of research and the development of clinical trial design
  • Educating patients and clinicians on early symptoms and the importance of genetic testing 
  • Encouraging a global initiative and ensuring registries are collecting data in a standardized manner 
  • Navigating strategies to ensure diversity and inclusion in clinical trials 
  • Empowering a “care triad” model for collaboration among patients and nephrologists

11:30 am
Workshop E: A Regulatory Perspective: Understanding the Approach Towards Selecting Efficacy Endpoints for Rare Disease Clinical Trials

Synopsis

Attend this workshop to better understand how to demonstrate clinically meaningful and statistically significant endpoints to identify the windows of opportunity for the future treatments of IgAN, FSGS and ADPKD.

  • Harmonizing global regulations to standardize endpoints for rare and genetic kidney diseases
  • Developing disease-specific endpoints for rare kidney diseases with different phenotypic presentations and disease pathology 
  • Navigating endpoints beyond total kidney volume and eGFR slope 
  • Understanding how to go about INDs and when to request a meeting 
  • Leveraging registries to aid in validating endpoints
  • Selecting endpoints for rare kidney disease clinical trials for efficient assessment of clinical signal for decision making in drug development

3:00 pm
Workshop F: Enhancing the Development of Curative Therapies by Understanding Payers’ Requirements When Kidney Diseases Are Rare

Synopsis

Unite at this workshop to apprehend how payers can facilitate access to care for patients with an urgent unmet medical need.

  • Discussing the current scope of insurance coverage for limited patient populations 
  • Deliberating the future of reimbursement strategies pre-launch 
  • What are the key challenges that both payers and industry are facing? 
  • Understanding the steps and diagnostics that are covered by insurance