Conference Day Two

Thursday 14th September 2023

8:55 am Chair’s Opening Remarks

Reimagining Endpoints to Demonstrate Efficacy in Shorter & More Efficient Clinical Trials

9:00 am Tackling the Challenges: Outlining a Novel Molecule for the Treatment of FSGS

Synopsis

  • Detailing a multicenter phase III FSGS trial and addressing the timeline challenges 
  • Improving the recruitment of patients and encouraging the involvement of paediatric patients
  • Navigating the route towards surrogate endpoint validation

9:30 am Developing a Roadmap to Address Increasing Patient Awareness, APOL1 Testing & Study Participation: Lessons for Genetic Kidney Disease Drug Development

Synopsis

  • Increasing disease awareness and genetic testing for APOL
  • Enhancing patient participation and awareness in clinical studies
  • Bringing the kidney community together to advance kidney health and address disparities

10:00 am Panel Discussion: A Global Initiative: Increasing Collaborative Work to Unite Towards a Streamlined Trials Process

  • Alex Dinh Clinical Leader, Translational & Experimental Medicine, Janssen Research & Development
  • Jeffrey Hafkin Senior Director, Otsuka

Synopsis

  • Agreeing on samples and linked clinical data to decipher the relationship between the biomarker and clinical outcomes 
  • Collecting patient databases by improving the global repository 
  • Including study coordinators to aid in prioritising measurements

10:30 am Morning Break & Networking

Better Recapitulating Kidney Diseases & Elucidating Next-Generation Diagnostic Techniques to Expedite Translational Success

11:30 am Panel Discussion: Deliberating the Benefits & Challenges of Current Preclinical Models to Move Towards Translational Success

  • Thomas Natoli Director - Pharmacology, Cardiovascular & Metabolic Disease, Janssen Research & Development
  • Rachel Gallagher Scientist, 4:59 NewCo, a 5AM Ventures Company

Synopsis

  • Increasing the relevance and accessibility of animal models to ease and shorten the drug discovery process 
  • How are rats and mice used in rare and genetic kidney diseases? 
  • Assessing the ability to recapitulate more than one aspect of disease

12:00 pm Unravelling the Opportunities Genomics Present in Delivering Precision Medicine in the Treatment of Rare Kidney Disorders

  • Eric Green Chief Scientific Officer, Maze Therapeutics

Synopsis

  • Summarizing the progress in genomic discovery in nephrology 
  • Tackling the translational challenges including both diagnosis and therapy 
  • An overview of the emerging platforms that may enable unexpected innovation and increased opportunity for patient level benefits

Innovating Clinical Trial Design by Ensuring the Evolvement of Registries & Enrolment to Improve Patient Access & Treatment

11:30 am Pooling Observational & Clinical Trial Data Sets to Evaluate Long-Term Outcomes

Synopsis

  • Combining data from a myriad of different studies and creating a cohort which can evaluate long-term outcomes across various subgroups of patients with rare conditions
  • Unravelling its clinical implications in rare and genetic kidney diseases 

12:00 pm Using Real-World Evidence to Increase Patient Diversity in Rare Disease Early Development Trials

  • Alex Dinh Clinical Leader, Translational & Experimental Medicine, Janssen Research & Development

Synopsis

  • Racial and ethnic minority populations have historically been underrepresented in rare disease early phase trials
  • Reducing barriers to clinical trial diversity is essential for health equity
  • Incorporation of real-world evidence into trial planning has the potential to increase recruitment and enrolment of diverse patient populations

12:30 pm Lunch Break and Networking

Discovering Novel Approaches to Trial Designs to Transform Current Studies for Rare & Genetic Kidney Disease

1:30 pm Harnessing Advanced Genome Sequencing to Diagnose Genetic Syndromes & Uncovering Underlying Pathophysiology

  • Ali Gharavi Professor - Medicine Research, Columbia University Medical Center

Synopsis

  • Incorporating genomic and molecular profiling in routine clinical practice to improve diagnosis and a new era of tailored therapies
  • Elucidating clinical and genetic characteristics of patients with rare diseases that manifest differently
  • Interrogating genome-wide association studies to advance understanding of pathophysiology and disease mechanisms

2:00 pm Chair’s Closing Remarks