Drug discovery company Proteostasis Therapeutics has completed the recruiting process for its Inaugural Cystic Fibrosis Clinical Advisory Board, a new board appointed by the company to act as a strategic resource responsible for the selection of upcoming studies of its compounds for treating cystic fibrosis.
The company is dedicated to developing pharmaceutical options for unmeet medical needs, especially regarding orphan and neurodegenerative diseases that cause abnormalities in protein folding, trafficking, and clearance. Its main focus has been enhancing the effectiveness of CF therapy, studying the combination of ivacaftor/lumacaftor in the gold-standard HBE cell assay for the most common mutation of the disease, F508del/F508del.
In its cystic fibrosis drug development efforts, Proteostasis Therapeutics uses its Disease Relevant Translation (DRT™) platform, which is a combination of the company’s proprietary screening approach together with state-of-the-art medicinal chemistry and functional disease-relevant cellular models in order to create selective drug candidates.
The members of the Proteostasis Clinical Advisory Board were chosen with the help of StratAcuity’s CBO Patrick Marshall, and includes the Professor Emeritus of Pediatrics at the Lucile Salter Packard Children’s Hospital at Stanford University, Richard B. Moss, MD, who was Chief of Pediatric Pulmonary and Allergy Divisions, and allergy-immunology and pulmonary fellowship director, and will now be the new Chairman of the Board. Marshall had already served as Director of the Cystic Fibrosis Center at Stanford, as well as Chair of the Protocol Review Committee and site principal researcher at the Cystic Fibrosis Therapeutics Development Network.
Jane C. Davies, MD, FRCPCH, a Professor of Pediatric Respirology and Experimental Medicine at Imperial College, London, was also appointed to the board. An Honorary Consultant in Pediatric Respiratory Medicine at the Royal Brompton & Harefield NHS Foundation Trust, Davies’ experience includes investigator-initiated and pharma-sponsored research, and she is a co-PI for the European CF Society Clinical Trials Network, where she is conducting the Lung Clearance Index Core facility.
The board will also include the expertise of Michael R. Knowles, MD, who currently serves as a Professor of Pulmonary and Critical Care Medicine at University of North Carolina (UNC), Chapel Hill. Knowles leads a study on genetic modifiers of disease phenotype (severity) in CF lung and liver disease, as well as a consortium study within eight sites dedicated to understanding the rare genetic disorders of mucociliary clearance.
Felix A. Ratjen, MD, PhD, the Chief of Pediatric Respiratory Medicine at The Hospital for Sick Children, and Professor of Paediatrics at The University of Toronto, and Senior Scientist at the Research Institute in the Department of Physiology and Experimental Medicine, was also chosen. The researcher is currently the co-leader of the Cystic Fibrosis Centre at The Hospital for Sick Children, as well as its Medical Director of the Clinical Research Unit.
Isabelle Sermet-Gaudelus, MD, PhD, a Professor of Pediatric Medicine at l’Hôpital Necker-Enfants Malades in Paris, France, and Pamela L. Zeitlin, MD, PhD, is Professor and Director of Pediatric Pulmonary Medicine and the Co-Director of the Cystic Fibrosis Center at Johns Hopkins University are the two other members of the new board. While the former has been investigating the modulation of the clinical severity of cystic fibrosis associated to fluid transference, the latter studies the role of chloride channels in inherited diseases of the respiratory tract on CF patients.