Our Scientific Advisory Board consists of a committed group of highly respected and experienced researchers, clinicians, and health industry leaders who provide guidance on strategic research priorities for Emily’s Entourage.
Chair, Department of Physiology; Isaac Ott Professor of Physiology, Perelman School of Medicine, University of Pennsylvania
J. Kevin Foskett, PhD, is the Isaac Ott Professor of Physiology and Chair of the Department of Physiology in the Perelman School of Medicine at the University of Pennsylvania. He is also Professor in the Department of Cell and Developmental Biology, and a member of the Mahoney Institute for Neurosciences and the Abramson Cancer Center. Dr. Foskett has been a stalwart member of Penn Medicine for nearly 20 years. After earning his doctorate at the University of California, Berkeley, he held positions at Toronto’s Hospital for Sick Children and at the University of Toronto before coming to Penn.
Dr. Foskett’s research for over 30 years has focused on the mechanisms and regulation of ion transport and signal transduction. He has had long standing interest in exocrine epithelial ion and water transport, with a particular focus on the genetic disease cystic fibrosis (CF), and has published extensively on various aspects of the biophysics and cell biology of CFTR and the cell biology of lung submucosal glands, the site of the primary defect in the lungs in CF. He served as a consultant to the Cystic Fibrosis Foundation in their collaboration with Aurora Biosciences/Vertex that directly led to the development of the first drug that targets the basic defect in CF.
Professor of Biochemistry/Molecular Biology at the Faculty of Sciences, University of Lisboa (Portugal) and Group Leader at the Biosystems & Integrative Sciences Institute (BioISl)
Margarida D. Amaral, PhD, is a Full Professor of Biochemistry/ Molecular Biology at the Faculty of Sciences, University of Lisboa (Portugal) and Group Leader at the Biosystems & Integrative Sciences Institute (BioISl). For over 20 years, her research career has been dedicated to the basic and translational research of cystic fibrosis (CF).
Dr. Amaral is an alumna of European Molecular Biology Laboratory and of the Gulbenkian Institute of Science. She is also a member of the European Molecular Biology Organization and the Portuguese Academy of Sciences. Dr. Amaral has received multiple awards, including the Pfizer-SCML Award for Basic Biomedical Research and the Annual Award of the European Cystic Fibrosis Society. The overall goal of Dr. Amaral’s research is to understand the molecular and cellular mechanisms of CF and to translate this knowledge into the benefit of patients. She has authored more than 170 international papers.
James C. and Elizabeth T. Lee Endowed Chair of Biochemistry; Professor and Chair of the Department of Biochemistry and Molecular Genetics in the Heersink School of medicine at the University of Alabama at Birmingham (UAB)
Dr. Bedwell serves as the Co-Director of the UAB Structural Biology Program and as Associate Director of the Gregory Fleming James Cystic Fibrosis Research Center. Dr. Bedwell is also an Editorial Board Member for the Journal of Biological Chemistry.
The major goal of Dr. Bedwell’s lab is to develop therapeutic strategies to treat genetic diseases caused by nonsense mutations. His team uses a combination of genomics, genetics, biochemistry, and cell biology to better understand the molecular details of translation termination in order to develop viable therapeutic strategies aimed at suppressing nonsense mutations and restoring the expression of full-length, functional proteins.
One disease currently under active investigation is cystic fibrosis (CF). Roughly 10% of CF patients carry a nonsense mutation in the CFTR gene. Dr. Bedwell’s group has participated in the identification of functionally distinct compounds that suppress termination at nonsense mutations in the CFTR gene and are examining these so-called “readthrough” agents in various experimental models. They have also identified the amino acids that become inserted during the suppression of many nonsense mutations in mammalian cells and are using that information to develop strategies to enhance the CFTR activity restored by nonsense suppression using CFTR correctors and potentiators.
Other diseases that Dr. Bedwell’s lab is using to study the utility of this approach include neurofibromatosis, FOXG1 deficiency, Rett syndrome, and Hurler syndrome. The ultimate goal of this approach is to provide an effective treatment for patients with a range of genetic diseases caused by nonsense mutations.
Professor, Microbiology and Immunology, Geisel School of Medicine at Dartmouth College, Associate Director, DartCF Research and Development Program in Cystic Fibrosis at Dartmouth
Dr. Bomberger has been active in the cystic fibrosis (CF) research community for over 15 years, participating in many national committees and leading local CF programs. She currently chairs the CF Foundation’s Infection Research Initiative and the Best Junior Investigator Basic Science Award at the annual NACFC, as well as serves on the NACFC program planning committee and research training study section for the CFF. Dr. Bomberger has mentored a number of CF scientists and physician scientists on CFF fellowships and transition awards and is dedicated to training the next generation of CF scientists.
Dr. Bomberger recently joined the faculty at the Geisel School of Medicine at Dartmouth, after starting her independent research program at the University of Pittsburgh. Utilizing her training in epithelial cell biology and microbiology as a foundation, the Bomberger laboratory studies host-pathogen interactions in the lung, focusing on the modulation of airway epithelial cell biology by respiratory pathogens and the pathogen’s response to the host. Her long-term research goals are to elucidate the cellular and molecular mechanisms whereby CF pathogens and respiratory viruses synergize to impact lung disease and, ultimately, identify new therapeutic approaches to control combined chronic P. aeruginosa and respiratory virus infections in people with CF.
Paul F Harron Jr Associate Professor of Medicine; Perelman School of Medicine University of Pennsylvania; Program Director, Adult Cystic Fibrosis Program; Member, Lung Transplantation Program; Physician Leader, Advanced Lung Disease Research Group
Denis Hadjiliadis, MD, did his undergraduate studies at McGill University and completed his medical degree at the University of Toronto in 1995. He subsequently pursued training in Internal Medicine at the Mayo Clinic and Pulmonary and Critical Care at Duke University, where he also completed a Master’s of Health Sciences in 2001. He joined the faculty at the University of Toronto in 2001, after further training in lung transplantation and cystic fibrosis (CF). He joined the University of Pennsylvania in 2005 and has been the Adult Program Director of the Cystic Fibrosis Program since 2009, while remaining active at the Penn Lung Transplant Program. He is currently the Paul F. Harron Jr. Associate Professor of Medicine, at the Perelman School of Medicine of the University of Pennsylvania.
Dr. Hadjiliadis has a very active practice in one of the largest adult cystic fibrosis programs in the country and also manages patients before and after lung transplantation, mainly with CF. In addition, he is the physician leader of the Advanced Lung Disease Research Group and has been the local Principal Investigator for many clinical trials. He also has been involved in many investigator initiated clinical trials in areas of susceptibility to Pseudomonas infection, inspiratory muscle strength and training in CF, use of flaxseed in CF and many others. He always has helped train many young physicians and foster their careers in CF.
Senior Lecturer in Molecular Physiology and Head of CF Gene Editing Lab, Department of Physiology; School of Medicine, University College Cork, Ireland
In 2012, the gene editing group led by Patrick Harrison, PhD, reported the first successful correction of a CFTR mutation in a cell model of cystic fibrosis. Since then, his lab has successfully applied a range of new strategies to correct a number of PTC and splicing mutations with greater efficiency, and with a focus on approaches that are amenable to clinical development. The lab is currently funded by the CF Foundation (USA), CF Trust (UK) and Science Foundation Ireland, and is collaborating with other groups in London, across Europe and North America towards the common goal of developing gene-based therapies suitable for everyone with CF.
Dr. Harrison has been a regular presenter and session organizer at European and American CF conferences since 2014, including a keynote talk on Gene Editing for CF: Heading Towards a Brave New World, at the 2017 European CF conference. Outside the lab, Dr. Harrison is the Chair of the CF Trust’s Research Grants Review Committee and European Editor for the journal Gene Therapy. He also supports research on other genetic diseases working with Cystinosis Ireland, and as a board member of The Irish Platform for Patients’ Organisations, Science and Industry.
Professor, Hebrew University of Jerusalem
Department of Genetics
Prof. Batsheva Kerem received her B.Sc. in Biology (1979) and her PhD (1986) from the Hebrew University. Her postdoctoral training was at the Hospital for Sick Children, Toronto Canada (1987-1990). Since 1990, she is a faculty member at the Department of Genetics, The Life Sciences Institute, The Hebrew University, Jerusalem, Israel. Prof. Kerem was appointed a full professor at 2003.
At the Hebrew University, she established and chaired the National Genomic Knowledge Center at the Institute of Life Sciences and served as the Head of Department of Genetics (2004-2006) and as the Head of the Authority for research students (from 2007-2011). From 2013, Prof. Kerem is the President advisor for promotion of women in science. Dr. Kerem received numerous prizes, including the Joels Senior Lectureship for Excellence in Science (1996), Teva Prize for Excellence in Human Genome (1993), Julodan Prize for Contribution to Medicine (1993), Abisch-Frenkel Prize for Excellence in Life Sciences (2003) and Emet Prize (2008). Dr. Kerem is a member of the European Molecular Biology Organization (EMBO). She serves on the editorial board of the European Journal of Human Genetics and EMBO Reports. Prof. Kerem has published over 120 papers.
Dr. Kerem has been working in cystic fibrosis (CF) research for already 29 years. She has been part of the group that cloned the CFTR gene. Through the years, she focused on two main CF research projects: development of therapeutic approaches for patients carrying nonsense mutations, including studies on cellular homeostatic systems affecting the response of patients to readthrough treatments. The other project focuses on developments of new therapeutic concepts for patients carrying splicing mutations/variants.
Maria P. Limberis, PhD, has more than 20 years of gene therapy research experience. She has dedicated her research career to the discovery and development of novel gene therapies for the potential treatment of cystic fibrosis (CF) airway disease. Most recently, Dr. Limberis served as Vice President of Research at Spirovant Sciences, Ltd. Previously, she was an Associate Professor in the Department of Medicine at the University of Pennsylvania, leading pre-clinical R&D and translational gene therapy programs in CF and airborne infectious diseases.
Through her doctoral research at the University of Adelaide and the Women’s and Children’s Hospital in Adelaide, Australia, Dr. Limberis developed a novel gene therapy regimen for lungs affected by CF, involving a compound found naturally in the lungs that ‘conditions’ the lungs to allow diseased airway cells to take up the therapeutic CF gene. The results, published in Human Gene Therapy in 2002, were the first to demonstrate that lentiviral-based gene therapy “corrects” the CF airway defect in an animal model of CF. This research has served as the foundation of many gene therapy studies, and is cited in more than 155 publications.
Medical Director, Clinical Research Services; Co-Director, Adult Cystic Fibrosis Program; CF Therapeutics Development Director; Professor (tenured), Departments of Medicine and Pediatrics, The Tuchman Family Division of Pulmonary, Critical Care and Sleep Medicine, National Jewish Health
Dr. Jennifer Taylor-Cousar is a tenured professor of adult and pediatric pulmonary medicine at National Jewish Health (NJH), where she serves as the Medical Director of Clinical Research Services. She oversees the care of children with pulmonary disease and adults with cystic fibrosis (CF). Dr. Taylor-Cousar is co-director of the Adult CF Program and Director of the CF Therapeutics Development Network (TDN) research that is conducted at NJH, and has been site primary investigator on more than 40 studies, and global site investigator on three studies. Her investigator initiated research focuses on the development and evaluation of novel therapies for the treatment of CF, and determination of the etiology and treatment of bronchiectasis in non-human primates.
She serves on numerous local and national committees including the CF Foundation’s Clinical Research Advisory Board, the CF TDN’s Clinical Research Executive Committee and as Chair of the CF TDN’s Women’s Health Research Working Group. She recently completed service on the American Thoracic Society (ATS) Scientific Advisory Committee, and was voted Chair-Elect for the 2019-2020 ATS Clinical Problems Programming Committee (Chair, 2020-2021). She has co-chaired numerous sessions and given invited lectures at the ATS International Conference, and the North American, European and Australian CF Conferences, as well as at regional CF and pulmonary conferences and national and international veterinary conferences. She is an active member of the Colorado Chapter CF Board.
Dr. Taylor-Cousar received her undergraduate degree in human biology from Stanford University in 1993. She completed her doctorate in medicine in 1998, combined residency in internal medicine and pediatrics in 2002, and her combined fellowship in adult and pediatric pulmonary medicine in 2006 at Duke University Medical Center. Dr. Taylor-Cousar obtained her Master of Clinical Science from the University of Colorado in 2015.
Chief Scientific Officer, BioMotiv; Board Chairman, Cystic Fibrosis Foundation Therapeutics, Inc.
Ted J. Torphy, PhD, is the Chief Scientific Officer for BioMotiv, the for-profit arm of The Harrington Project, the mission of which is to accelerate the translation of academic discoveries into medicines. Since 2004, Dr. Torphy has also chaired the board of the Cystic Fibrosis Foundation Therapeutics, Inc. During this period the Foundation introduced the venture philanthropy model of co-investing with biotechnology and pharmaceutical companies to bring breakthrough therapies to individuals with cystic fibrosis (CF), including KALYDECO® and ORKAMBITM, the first treatments that correct the fundamental defect causing CF.
Before joining BioMotiv Dr. Torphy spent 30 years in the pharmaceutical industry, working at SmithKline Beecham and Johnson & Johnson. His focus was on drug discovery and non-clinical development of both small molecules and biologics in the Cardiovascular, Metabolic, Pulmonary, Renal and Inflammatory Diseases therapeutic areas. During this period he was instrumental in the discovery and development of multiple marketed products, including SIMPONI®, STELERA® and SYLVANT®. Dr. Torphy holds a B. degree in Pharmacy from the University of Wisconsin and a PhD in Pharmacology and Toxicology from West Virginia University. He completed his postdoctoral training at the University of California, San Diego.
Executive Vice President, The Wistar Institute; Director, Wistar Vaccine Center; W.W. Smith Charitable Trust Professor in Cancer Research
Preeminent immunologist and vaccine expert, David B. Weiner, PhD, is executive vice president of The Wistar Institute, director of the Wistar Vaccine Center, and the W. W. Smith Charitable Trust Professor in Cancer Research. Dr. Weiner directs a translational research laboratory in the area of Molecular Immunology. His group is one of the pioneering research teams in establishing the field of DNA vaccines and immune therapies. Important reports from his lab include the first DNA vaccine studied for HIV as well as for cancer immune therapy, the early development of DNA encoded genetic adjuvants including the particularly relevant IL-12, advances in gene optimization, and advances in electroporation technologies resulting in improved gene delivery among others. His group worked with collaborators to become the first to move DNA technology into human study. His laboratory’s work helped revitalize the field through advancement of new synthetic DNA design and modification of EP delivery approaches resulting in potent immune induction as well as the first successful Phase IIb DNA efficacy study (for HPV immune therapy) in humans.
Weiner is the recipient of numerous honors including election as a fellow to both the American Association for the Advancement of Science in 2011 and the International Society for Vaccines in 2012. He is the recipient of the NIH Director’s Transformative Research Award and received the Vaccine Industry Excellence Award for Best Academic Research Team in 2015 at the World Vaccine Congress. Weiner was honored with the prestigious Hilleman Lectureship in 2015 at the Children’s Hospital of Philadelphia Grand Rounds session and received a Stone Family Award from Abramson Cancer Center for his groundbreaking work on DNA vaccines for cancer immune therapy.
In March 2016, Weiner returned to Wistar from his position at The University of Pennsylvania School of Medicine as professor of Pathology and Laboratory Medicine. From 1990 to 1993, Weiner held a joint position as assistant professor of Pathology and Laboratory Medicine at The Wistar Institute and the University of Pennsylvania School of Medicine.
Weiner graduated with a BS in biology from SUNY at Stony Brook, in Stony Brook, NY, a MS in biology from the University of Cincinnati and a PhD in developmental biology from the University of Cincinnati College of Medicine.
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