Proteostasis Creates Cystic Fibrosis Clinical Advisory Board of Experts

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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Specialty drug discovery company Proteostasis Therapeutics, Inc. has officially welcomed 6 new members to its clinical advisory board, who will have a direct impact on the company’s efforts in developing therapies for cystic fibrosis. The company is internationally known for its focus on developing cutting edge treatments for diseases caused by aberrations in protein folding, trafficking, and clearance.

The new clinical advisory board will be an essential, authoritative source for the upcoming selection and research on some of the company’s flagship compounds, which have shown promising results in enhancing the activity of combined ivacaftor and lumacaftor in today’s gold-standard HBE cell assay for F508del — the most common genetic mutation in cystic fibrosis patients.

“We are honored that these world renowned cystic fibrosis clinicians have agreed to bring their expertise to form our team of clinical advisors,” said Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics. “The knowledge and experience that our inaugural clinical advisory board members bring to Proteostasis will be invaluable not only in the selection process of which of our proprietary PTI CFTR compounds should proceed to clinical development, but also in providing guidance on the design and conduct of the future clinical trials of selected drug candidates.”

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The six newly-appointed clinical advisors are the following:

  • Dr. Richard B. Moss, MD – Professor Emeritus of Pediatrics at the Lucile Salter Packard Children’s Hospital at Stanford University. He will be the first Chairman of the Clinical Advisory Board.
  • Dr. Jane C. Davies, MD, FRCPCH – Professor of Pediatric Respirology and Experimental Medicine at Imperial College, London and an Honorary Consultant in Pediatric Respiratory Medicine at the Royal Brompton & Harefield NHS Foundation Trust.
  • Dr. Michael R. Knowles, MD – Professor of Pulmonary and Critical Care Medicine at University of North Carolina (UNC), Chapel Hill.
  • Dr. Felix A. Ratjen, MD, PhD – Chief of Pediatric Respiratory Medicine at The Hospital for Sick Children, Professor of Pediatrics at The University of Toronto, and Senior Scientist at the Research Institute in the Department of Physiology and Experimental Medicine. He co-leads the Cystic Fibrosis Centre at The Hospital for Sick Children, and is the Medical Director of its Clinical Research Unit.
  • Dr. Isabelle Sermet-Gaudelus, MD, PhD – Professor of Pediatric Medicine at l’Hôpital Necker-Enfants Malades in Paris, France. She has developed and conducted several therapeutic trials for cystic fibrosis for both academic and company-initiated investigations.
  • Dr. Pamela L. Zeitlin, MD, PhD – Professor and Director of Pediatric Pulmonary Medicine and the Co-Director of the Cystic Fibrosis Center at Johns Hopkins University. Her research focuses on the role of chloride channels in inherited diseases of the respiratory tract, in particular, cystic fibrosis.

In particular, Dr. Moss, the new Chairman of the Clinical Advisory Board, believes his primary and extensive experience as a pediatric pulmonologist will prove essential in developing novel treatments for patients with cystic fibrosis, as the disease is frequently diagnosed in children. Cystic fibrosis patients are estimated to live up to an average of 40 years old, before respiratory failure, a complication from a severe, opportunistic respiratory infection, or a gastrointestinal complication causes mortality. He looks forward to being an active member of the board as he knows he will learn from the company as much as it will learn from his expertise.