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Biomarker for Rare Diseases Summit

Biomarker for Rare Diseases Summit


Date of beginning

Tuesday, 09 November 2021


2 days




Charlotte Harrison


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As reports predict the global biomarker test market will exceed $16bn in coming years, the ability to identify and utilize predictive, diagnostic and prognostic biomarkers is a priority for drug developers. In rare disease, affecting over 400 million worldwide and where clinical insights exist for only around 400 of the 7000+ disease identified, biomarkers are a valuable tool to accelerate drug development where there is current great unmet need. With this, we are excited to announce the launch of the inaugural Biomarkers for Rare Diseases Summit. Uniting world leaders developing novel therapeutics for rare disease, we will discuss learnings, challenges and future directions at the only summit dedicated to approaches to identify, test and proof clinical biomarkers in rare disease for regulatory approval. From identifying disease driving biomarkers, robust assay validation, harnessing latest technologies and demonstrating therapy efficacy, we bring you the definitive platform to accelerate bench to bedside rare disease development towards regulatory filing and patient populations. - Explore the Characterization and Identification of Disease Driving BiomarkersConsidering the genotypic and phenotypic variance of disease, discuss approaches and hurdles to identify and discover biomarkers, and how this informs future data collection and study design with Vifor Pharma, Center for Genomic Interpretation, NIH, Regulus Therapeutics and Boston Children's Hospital - Design Robust Biomarker Validation Plans for Meaningful ConclusionsWith the variability of biomarkers over time and assay analytical validation a significant hurdle, we will hear progress and strategies to better define biomarkers and support their use as a drug development tool from the likes of Novo Nordisk and Foundation for the National Institutes of Health Demonstrate the Clinical Utility of Biomarkers for Rare Disease Drug DevelopmentFrom retrospective and prospective data informing patient identification for clinical trials to novel natural history study design, hear exclusive clinical case studies and insights from Diamyd, Triplet Therapeutics and CHDI Foundation. - Show Therapy Efficacy Demonstration in Preparation for Regulatory FilingAs regulatory agencies endorse the need for flexibility in the review process, consider how biopharma is approaching the definition of acceptable endpoints and discuss navigating regulation pathways and a potential future roadmap for the approval of your pipelines with Takeda and Foundation for the National Institutes of Health - Detect and Diagnose Disease Earlier Harnessing Emerging Techniques and TechnologiesBreakthroughs in technology are driving industry's ability to diagnose disease earlier, investigate pathophysiology and identify new drug targets, hear expert opinions on technologies you should consider as well as their cost and practical application from Rady Children's Institute for Genomic Medicine, Johnson and Johnson and Passage Bio URLs:Tickets: Brochure:  Date and Time: On Tue, Nov 9, 2021 (9:00 AM) - Wed, Nov 10, 2021 (3:00 PM) Price:2 Day Conference: USD 1799.00 Speakers: Anka G. Ehrhardt Director Clinical Research CHDI Foundation, Julie Eggington Co-Founder and Chief Executive Officer Center for Genomic Interpretation, Ulf Hannelius President and Chief Executive Officer Diamyd Medical, Eline Appelmans Director of Neuroscience Research Partnerships Foundation for the National Institutes of Health, Wesley Horton Scientific Project Manager Foundation for the National Institutes of Health, Mark Kiel Chief Science Officer and Co- Founder Genomenon, Carlo Brugnara Director, Hematology Lab Professor of Pathology Harvard Medical School, Robert Neusner Global Strategy Director, Precision Medicine Johnson and Johnson, Jaydira Del Rivero MD National Cancer Institute, NIH, Matthias Von Herrath Vice President and Senior Medical Officer, Global Chief Medical Office Novo Nordisk, Yan Ni Executive Director of Biomarkers and Precision Medicine Passage Bio, Stephen Kingsmore President and Chief Executive Officer Rady Children's Institute for Genomic Medicine, Edmund Lee Executive Director, Biology Regulus Therapeutics, Katherine Call Former Senior Director, Head of Proteogenomics Sanofi Genzyme, Robert Latzman Associate Director, Quantitative Sciences Takeda, Peter Bialek Head of Translational Biology Triplet Therapeutics, Domenico Merante Global Clinical Development TA Lead Nephrology and Orphan Disease Area Vifor Pharma