2021 Expert Speakers

Speakers

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Anka G. Ehrhardt
Director Clinical Research
CHDI Foundation

Day Two

Wednesday, November 10 2021

2:30 pm | Panel Discussion: Choosing the Right Technology for Your Biomarker Investigations

10:30 am | Rare & Precious: Strategic Collection & Use of Clinical Samples The largest currently available natural history study in Huntington’s disease illustrates key motivations, concepts, and considerations in building and using clinical sample collection to drive research in rare diseases:

Julie Eggington
Co-Founder & Chief Executive Officer
Center for Genomic Interpretation

Day One

Tuesday, November 9 2021

10:00 am | Cohort Confounders: Inaccuracies in Genetic Tests & What to Do About It

Ulf Hannelius
President & Chief Executive Officer
Diamyd Medical

Day Two

Wednesday, November 10 2021

9:30 am | Patient Identification for Clinical Trials – The Case of HLA & Antigen-Specific Immunotherapy in Type 1 Diabetes

Eline Appelmans
Director of Neuroscience Research Partnerships
Foundation for the National Institutes of Health

Day Two

Wednesday, November 10 2021

12:30 pm | Panel Discussion: Navigating Rare Disease Drug Development Regulation Pathways

Wesley Horton
Scientific Project Manager
Foundation for the National Institutes of Health

Day One

Tuesday, November 9 2021

3:45 pm | Consortia-based Analytical Validation of Assays for Drug Development

Mark Kiel
Chief Science Officer & Co- Founder
Genomenon

Day Two

Wednesday, November 10 2021

3:45 pm | Chair’s Closing Remarks, End of Day Two & Networking

12:30 pm | Panel Discussion: Navigating Rare Disease Drug Development Regulation Pathways

9:20 am | Chair’s Opening Remarks

Carlo Brugnara
Director, Hematology Lab Professor of Pathology
Harvard Medical School

 

Day One

Tuesday, November 9 2021

1:00 pm | Biomarkers in Sickle Cell Disease & Their Application to Gene Therapy

Robert Neusner
Global Strategy Director, Precision Medicine
Johnson & Johnson

Day Two

Wednesday, November 10 2021

2:30 pm | Panel Discussion: Choosing the Right Technology for Your Biomarker Investigations

Jaydira Del Rivero
MD
National Cancer Institute, NIH

Day One

Tuesday, November 9 2021

12:00 pm | Biomarker Identification in Rare Neuroendocrine Cancer

Day Two

Wednesday, November 10 2021

2:30 pm | Panel Discussion: Choosing the Right Technology for Your Biomarker Investigations

Matthias Von Herrath
Vice President & Senior Medical Officer, Global Chief Medical Office
Novo Nordisk

 

Day One

Tuesday, November 9 2021

2:30 pm | Longitudinal Monitoring of Immune Biomarkers for Reliable Readouts

Yan Ni
Executive Director of Biomarkers & Precision Medicine
Passage Bio

Day Two

Wednesday, November 10 2021

2:30 pm | Panel Discussion: Choosing the Right Technology for Your Biomarker Investigations

Stephen Kingsmore
President & Chief Executive Officer
Rady Children’s Institute for Genomic Medicine

Day Two

Wednesday, November 10 2021

2:00 pm | Diagnosing Rare Disease & Identifying Drug Targets at Earlier Time Points

Edmund Lee
Executive Director, Biology
Regulus Therapeutics

Day One

Tuesday, November 9 2021

12:30 pm | Results From the First Cohort of Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Katherine Call
Former Senior Director, Head of Proteogenomics
Sanofi Genzyme

Day One

Tuesday, November 9 2021

4:15 pm | Chair’s Closing Remarks, End of Day One & Networking

9:20 am | Chair's Opening Remarks

Day Two

Wednesday, November 10 2021

2:30 pm | Panel Discussion: Choosing the Right Technology for Your Biomarker Investigations

Robert Latzman
Associate Director, Quantitative Sciences
Takeda

Day Two

Wednesday, November 10 2021

12:00 pm | Quantitative Approaches to Advancing Endpoint Development – With a Specific Focus on Cognition

Peter Bialek
Head of Translational Biology
Triplet Therapeutics

Day Two

Wednesday, November 10 2021

12:30 pm | Panel Discussion: Navigating Rare Disease Drug Development Regulation Pathways

10:00 am | Using Longitudinal Data to Understand How a Biomarker Behaves

Domenico Merante
Global Clinical Development TA Lead Nephrology & Orphan Disease Area
Vifor Pharma

Day One

Tuesday, November 9 2021

9:30 am | Clinically Meaningful & Feasibility of Some Selected Biomarkers in ANCA-Associated Vasculitis (MPA and GPA) for Clinical Development of Future Therapies

Day Two

Wednesday, November 10 2021

12:30 pm | Panel Discussion: Navigating Rare Disease Drug Development Regulation Pathways

Mathias Leinders
Associate Director, Clinical Development Neurology
Ionis Pharmaceuticals

Day One

Tuesday, November 9 2021

3:00 pm | Interactive Roundtable Discussion: ​Interactive Roundtable: New Solutions for Old Problems. Can Non-Invasive Biomarkers be the Clue?

Tom Defay
Deputy Head Diagnostics Strategy and Development
Alexion, AstraZeneca Rare Disease

Day Two

Wednesday, November 10 2021

3:15 pm | Enhancing Rare Disease Diagnosis Through Genome Sequencing and Genetics

Craig Martin
Chief Executive Officer
Global Genes

Day One

Tuesday, November 9 2021

10:30 am | Panel Discussion: Equity, Diversity & Inclusion in the Rare Disease Healthcare Community

Teneasha Washington
Assistant Professor
University of Alabama at Birmingham

Day One

Tuesday, November 9 2021

10:30 am | Panel Discussion: Equity, Diversity & Inclusion in the Rare Disease Healthcare Community

Rebecca Stewart
Chief Executive Officer
Rare Revolution Magazine

Nicola Miller
Editor in Chief
Rare Revolution Magazine

Day One

Tuesday, November 9 2021

10:30 am | Panel Discussion: Equity, Diversity & Inclusion in the Rare Disease Healthcare Community