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  5. Developing Alpha-1 Antitrypsin Therapeutics; Public Workshop - Agenda
  1. Workshops, Meetings & Conferences (Biologics)

Developing Alpha-1 Antitrypsin Therapeutics; Public Workshop - Agenda

Center for Biologics Evaluation and Research/FDA
National Heart Lung and Blood Institute/NIH
Alpha-1 Foundation

September 16, 2019 8:00 AM-5:00 PM
Natcher Auditorium, NIH Campus

8:00 - 8:30 AM Breakfast Sponsored by the Alpha-1 Foundation
8:30 - 8:40 AM Welcome and Introductory Remarks Peter Marks, MD, PhD
Director
FDA/CBER

NHLBI Representative

Miriam O’Day
CEO, Alpha-1 Foundation
8:40 - 9:15 AM Alpha-1 Antitrypsin Deficiency and Augmentation Therapy Robert Sandhaus, MD, PhD
Professor of Medicine
National Jewish Health
9:15 - 9:45 AM The Alpha-1 Foundation Scientific Infrastructure Charlie Strange, MD
Professor of Pulmonary and Critical Care Medicine
Medical University of South Carolina

Miriam O’Day
9:45 - 10:15 AM Update and Summary of Alpha-1 Therapeutics Ross Pierce, MD
Medical Officer
Division of Clinical Evaluation & Pharm/Tox
Office of Tissues and Advanced Therapies
FDA/CBER
10:15 - 10:30 AM Break
10:30 - 11:15 AM New Approaches to Clinical Trials John Scott, PhD
Director, Division of Biostatistics
Office of Biostatistics and Epidemiology
FDA/CBER
11:15 - 11:30 AM The Patient Journey Peggy Iverson
Alpha-1 Patient
11:30 - 12:00 PM Novel Targets for Alpha-1 Antitrypsin Adam Wanner, MD
Professor of Medicine
University of Miami School of Medicine
12:00 - 1:00 PM Lunch Sponsored by the Alpha-1 Foundation
1:00 - 2:15 PM Novel therapeutic Approaches that Raise Levels of Alpha-1 Antitrypsin Terence R. Flotte, MD
Professor of Medical Education
The University of Massachusetts Medical School

Stefan Marciniak, PhD
Professor of Respiratory Science
Cambridge Institute for Medical Research

Mark Brantly, MD
Professor of Medicine, Molecular
University of Florida College of Medicine

Klaus Wagner, MD, PhD
Chief Medical Officer
Inhibrx

Charlotte McKee, MD
Vice President, CF Clinical Development
Vertex Pharmaceuticals
2:15 - 2:40 PM Implications for Other Plasma Based Therapies Senator Rick Santorum
Patient Advocate
Partner in Plasma Technologies
2:40 - 3:00 PM Break
3:00 - 3:30 PM Therapeutic Approaches to Liver Disease in AATD James Hamilton, MD, MBA
VP, Clinical Development
Arrowhead Pharmaceuticals
3:30 - 3:50 PM Clinical Endpoints and Biomarkers in COPD Trials Jeanine D’Armiento, MD, PhD
Professor of Medicine
Columbia University
3:50 - 4:10 PM CT Densitometry Ken Chapman, MD
Director, Asthma & Airway Centre
University Health Network
4:10 - 4:45 PM Panel Discussion: Current Needs in Alpha-1 Antitrypsin Drug Development Moderated by Peter Marks
4:45 - 5:00 PM Summary and conclusions/next steps Peter Marks

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