Location: Digital

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 Biomarkers

Considering 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 & Boston Children’s Hospital

Design Robust Biomarker Validation Plans for Meaningful Conclusions

With 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 & Foundation for the National Institutes of Health

Demonstrate the Clinical Utility of Biomarkers for Rare Disease Drug Development

From 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 & CHDI Foundation.

Show Therapy Efficacy Demonstration in Preparation for Regulatory Filing

As 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 & Foundation for the National Institutes of Health

Detect & Diagnose Disease Earlier Harnessing Emerging Techniques and Technologies

Breakthroughs 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 & Johnson & Passage Bio

LEARN MORE