Independent Steering Committee

The CF RIA program is delighted to welcome an independent steering committee of external experts, both clinicians and research scientists in the field of CF, to review and assess all submitted CF RIA applications and award the research grants.

CHAIR - Bonnie Ramsey, MD

Dr Ramsey is the Director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute and has led the CF Therapeutics Development Network for over 16 years. She is Professor and Vice Chair for Research in the Department of Pediatrics and holds the Endowed Chair in Cystic Fibrosis at the University Of Washington School Of Medicine.

Dr Ramsey also works as an attending physician at Seattle Children’s Hospital. During her career, Dr Ramsey has focused on clinical care and research in the field of CF and is internationally recognized for her work in developing new therapies for patients with CF. A pioneer in aerosolized antibiotic usage in CF (TOBI), she has been a key investigator in the development of many CF therapies.

As co-principal investigator (PI) of the University of Washington Institute for Translational Health Sciences, she has helped set up an extensive clinical research infrastructure at Seattle Children’s Hospital. She is also the PI for a P30 for cystic fibrosis research from the NIDDK. Her current research interests lie in translational research, especially in the field of CF. Dr Ramsey is member of the American Pediatric Society and the Association of American Physicians. She is interested in the ethics of pediatric clinical research and has served on two Institute of Medicine committees focused on this topic.


Patrick Flume, MD

Dr Patrick Flume is currently Professor of Medicine and Pediatrics at the Medical University of South Carolina (MUSC). He is also the Director of the MUSC CF Center in Charleston, which oversees the largest adult CF program in South Carolina. 

The center provides state-of-the-art care to patients and families with CF, serves as primary educator of clinicians and basic scientists in the field of CF, and performs basic and clinical research in the field. Dr Flume also serves as the Powers-Huggins Endowed Chair for Cystic Fibrosis. Although Dr Flume continues to practice general pulmonary and critical care medicine, his primary interests are in CF, bronchiectasis, and non-tuberculous mycobacteria. He has an active clinical research program and chairs the advisory committee for the MUSC General Clinical Research Center. He is also engaged in the MUSC Clinical and Translation Science Award (CTSA) program, serving as the Program Director for the Research Center, a member of the Executive Committee for the CTSA, and Chair of the Scientific Review Committee.

In addition to this, Dr Flume also directs the Cardiopulmonary Exercise Laboratory and the MUSC Pulmonary Rehabilitation Program. During the course of his career, Dr Flume has worked on numerous grants and has authored or peer reviewed over 100 articles. Recognized by the national and international CF community, he currently serves on the CF Foundation Center Committee, Adult Care Consensus Committee, Advisory Task Force on Adult Issues, and Professional Education Committee. He also currently co-chairs the Pulmonary Practice Guidelines Committee.


Felix Ratjen, MD, FRCP(C)

Dr Felix Ratjen is the Division Chief of Pediatric Respiratory Medicine at The Hospital for Sick Children, Professor of Pediatrics at The University of Toronto, and Head of the Department of Translational Medicine at the Research Institute. He holds the Sellers Chair in Cystic Fibrosis from the Sick Kids Foundation, co-leads the CF center at Sick Kids and is Medical Director of the Clinical Research Unit.

Dr Ratjen is currently conducting multiple national and international clinical trials addressing CF lung disease. His research includes investigating new therapeutic strategies to target underlying defects, treatment of airway infections such as first infection with Pseudomonas aeruginosa, airway inflammation and other important aspects of the disease. In addition, he is involved in developing and validating new outcome measures to quantify important aspects of CF lung disease that can be utilized in clinical trials. In recent years Dr Ratjen has developed an interest in other lung disease, specifically in the clinical evaluation of hereditary hemorrhagic telangiectasia, for which he is leading one of the largest dedicated pediatric clinics.  

A member of several editorial boards including the American Journal of Respiratory and Critical Care Medicine, Thorax, Pediatric Pulmonology and the Journal of Cystic Fibrosis, Dr Ratjen works on multiple grant review panels. He is also a member of the organizing committees for the major respiratory meetings (American Thoracic Society, European Respiratory Society, and the North American CF conference).



Jeffrey Wine, PhD

Dr Jeffrey Wine is a Benjamin Scott Crocker Professor of Human Biology and the director of the Cystic Fibrosis Research Laboratory at Stanford University, California. Dr Wine received his bachelor’s degree in psychology from the University of Pittsburgh in 1965 and his PhD in physiological psychology from UC Los Angeles in 1971. In 1986 he was named a Professor in the Department of Psychology at Stanford University, a position that he still holds today. Dr Wine also sits on the CF Foundation Research & Research Training Review Panel. 

As director of the CF Research Laboratory at Stanford University, Dr Wine’s goal is to understand how a defective ion channel leads to CF, with an emphasis on ion channels and ion transport involved in gland fluid transport. His laboratory developed the methodology to study individual intact bronchial glands and have now expanded this methodology to the study of sweat glands in vivo in order to track the effects of CFTR-directed therapies. They were also the first to demonstrate that airway glands, which produce antibiotic-rich mucus that helps protect the airways, display a profound secretory defect in CF patients.

His current research is two-fold:

1) To further understand the role of submucosal glands in protecting the airways from infections; and how the loss of CFTR affects those functions.

2) To develop and refine biomarkers dedicated to increasing the speed with which CFTR-directed therapeutics can reach patients.


Margarida D. Amaral, PhD

Margarida Amaral is Full Professor of Biochemistry/Molecular Biology at the Faculty of Sciences, University of Lisboa (Portugal) and Coordinator of BioISI – the Biosystems and Integrative Sciences Institute. Professor Amaral is alumna of the European Molecular Biology Laboratory (2008–2010; 2016) and Gulbenkian Institute of Science (1983–1993).

Professor Amaral's research interests include the molecular and cellular mechanisms of CF. Her lab's major focus is the molecular and cellular mechanisms of biogenesis, traffic, and degradation of normal and mutant protein CFTR, which when mutated causes CF. 

Author of 116 international publications (average citations per article: 17.93), Professor Amaral is a member of the European Molecular Biology Laboratory (EMBO), the Portuguese Academy of Sciences (2014), and received the Pfizer Award for Basic Biomedical Research (2013) and the Annual Award of the European Cystic Fibrosis Society (2010). Professor Amaral is an elected member of the board of the European Cystic Fibrosis Society and Associate Editor of the Journal of Cystic Fibrosis and Scientific Reports.


Michael Knowles, MD

Dr Michael Knowles is Professor of Pulmonary and Critical Care Medicine at the Marsico Lung Institute and Cystic Fibrosis Research Center at the University of North Carolina (UNC). He is also former Director of the UNC Adult CF Clinical Center.

He has over 3 decades of clinical research experience that spans the disciplines of biology, physiology, and clinical trial design in both the academic and private sectors. His current research interests lie in mucociliary clearance, including primary ciliary dyskinesia; the identification of genes that modulate the severity of the pulmonary phenotype, pseudohypoaldosterism, and modifier genes in CF liver disease.

Dr Knowles is currently heading two large multicenter studies: 1) Genetic modifiers of disease phenotype (severity) in CF lung and liver disease, which also includes a recently formed international consortium doing a whole genome scan; and 2) a consortium with 8 sites in North America to study rare genetic disorders of mucociliary clearance.



Professor Alan Smyth is Professor of Child Health at the University of Nottingham and Honorary Consultant in Pediatric Respiratory Medicine at Nottingham University Hospitals NHS Trust. His major research interests are in novel ways of treating infection in CF and strategies to detect and minimize the adverse effects of treatment.

He has led a James Lind Alliance Priority Setting Partnership which worked with clinicians and patients to decide the top 10 priorities for clinical research in CF. Professor Smyth is Coordinating Editor of the Cochrane Cystic Fibrosis & Genetic Disorders Group, and Joint Editor-in-Chief of Thorax journal. When not working, he is a keen cyclist and pilot.


PrivacyTerms of Use
© 2019 Vertex Pharmaceuticals Incorporated | GL-20-1800008 | 02/2019
Vertex and the Vertex triangle logo are trademarks of Vertex Pharmaceuticals Incorporated 
CF RIA program funding is supported by Vertex Pharmaceuticals Incorporated