Working Group on Pediatric Gene Therapy & Medical Ethics Members
Members of the Working Group on Pediatric Gene Therapy and Medical Ethics (PGTME) come from a wide variety of fields, including industry, patient advocacy, medicine, law, and bioethics. This breadth of expertise is essential to advancing the mission of PGTME.
Alison Bateman-House, PhD, MPH, MA, Co-Chair
Dr. Bateman-House is an assistant professor in the Department of Population Health at NYU Langone. Within the department’s Division of Medical Ethics, she also co-chairs its Working Group on Compassionate Use and Preapproval Access (CUPA), whose members study ethical issues concerning access to investigational medical products. Dr. Bateman-House is co-chair of the Compassionate Use Advisory Committees (CompAC), which advise Janssen Pharmaceuticals, Inc., on the ethical allocation of its investigational products. She publishes and speaks extensively on access to investigational drugs and on the history and ethics of using humans as research subjects.
Lesha D. Shah, MD, Co-Chair
Dr. Shah is an assistant professor of psychiatry and the medical director of Child, Adolescent and Family Services at the Icahn School of Medicine at Mount Sinai, with dual board certification in psychiatry and child and adolescent psychiatry. Her primary academic focuses are pediatric research ethics and physician perspectives around medical decision-making. She studies issues of consent as they interface with innovative medicine, developmental disability, and family complexity. She is dedicated to medical education across the undergraduate to graduate medical education continuum and serves on the institutional review board at Mount Sinai.
Cara Hunt, MA, Project Manager
Ms. Hunt is a research associate in the Division of Medical Ethics at NYU Langone's Department of Population Health. She earned her undergraduate degree in science, technology, and society at Vassar College, after which she taught robotics and engineering to K-5 students. She then worked in the Education Department at Cooper Hewitt, Smithsonian Design Museum, during which time she earned a master's in bioethics at New York University.
Katherine Beaverson, MS
Ms. Beaverson is the patient advocacy lead for the Rare Disease Research Unit at Pfizer Inc., guiding the strategic planning and implementation of collaborations with patient advocacy groups to integrate patient expertise into early medicines research and development. A professionally trained genetic counselor, she spent 10 years at both New York Hospital-Weill Cornell Medical Center and Memorial Sloan-Kettering Cancer Center. She received her BA from Swarthmore College and her MS in human genetics from Sarah Lawrence College.
Jennifer deSante-Bertkau, MD
Dr. deSante-Bertkau is a pediatrician and clinical ethicist who attended Princeton University as an undergraduate and graduated from University of Pennsylvania School of Medicine with a combined doctor of medicine and master’s of bioethics degree. Upon completing her residency at the Children's Hospital of Philadelphia, she worked as a postdoctoral fellow in the National Institutes of Health Department of Bioethics. As an assistant professor at Cincinnati Children’s Hospital Medical Center, she is a hospitalist and medical educator.
Arthur Caplan, PhD
Dr. Caplan is the Drs. William F. and Virginia Connolly Mitty Professor of Bioethics in the Department of Population Health at NYU Langone and founding head of its Division of Medical Ethics. He co-chairs the Compassionate Use Advisory Committees (CompAC) and is the author or editor of 32 books and more than 750 papers in peer-reviewed journals. His most recent books are Getting to Good: Research Integrity in the Biomedical Sciences, with Barbara Redman, and Vaccination Ethics and Policy, with Jason Schwartz. During the 2019 coronavirus disease (COVID-19) pandemic, he is co-directing a new advisory group on sports and recreation for the U.S. Conference of Mayors, creating a working group on vaccine challenge studies, developing an ethical framework for distributing drugs and vaccines for Johnson & Johnson, and setting policy with the Western Institutional Review Board for research studies.
Moshe Cohn, MD
Dr. Cohn is a clinical assistant professor in the Department of Pediatrics at NYU Langone. Based at Hassenfeld Children’s Hospital at NYU Langone, he is board-certified in pediatric critical care and hospice and palliative medicine, and is a certified healthcare ethics consultant. Dr. Cohn teaches medical ethics at NYU Grossman School of Medicine and affiliated training programs. He is interested in the intersection of Jewish and secular medical ethics in pediatrics, brain death, end-of-life care, palliative ethics, and organ donation.
Timothy Cripe, MD, PhD
Dr. Cripe is the chief of pediatric hematology and oncology and blood and marrow transplantation at Nationwide Children’s Hospital in Columbus, OH, and a professor of pediatrics at the Ohio State University. His research focuses on leveraging viruses to develop novel therapeutic approaches to pediatric cancers. He has published more than 130 manuscripts, served on numerous national and international committees, and is a former chair of the U.S. FDA’s Advisory Committee for Cellular, Tissue, and Gene Therapies.
Rafael Escandon, PhD, DrPH, MPH
Dr. Escandon is the senior vice-president of medical affairs, policy, and patient engagement at BridgeBio Pharma, Inc., in San Francisco. He has worked in global clinical research and development and patient advocacy in the biotechnology industry for 25 years. His approach is to be highly considerate of the ethical implications of conducting and participating in clinical research, especially in rare diseases and in vulnerable and developing-world populations. He has served as adjunct faculty at the University of California Berkeley’s Clinical Research Management Program and as an educator in human rights and justice.
Richard S. Finkel, MD
Dr. Finkel is a pediatric neurologist who directs the Center for Experimental Neurotherapeutics at St. Jude Children's Research Hospital in Memphis, TN. His clinical practice and research interests have focused on neuromuscular and neurometabolic disorders. Dr. Finkel has participated in numerous clinical trials and natural history studies, and has contributed to the development of standard-of-care guidelines, outcome measures, and clinical trial design. He has published extensively and participates in a variety of working groups dedicated to the ethical delivery of healthcare for rare pediatric neurologic diseases.
Mr. Fischer serves as the chief advocacy officer for Parent Project Muscular Dystrophy (PPMD) and has served the organization in various capacities since 2004. PPMD is the leading organization in the fight to end Duchenne muscular dystrophy. Within PPMD, Mr. Fischer oversees all mission-related programming, including the organization's advocacy, care and research strategies. He also co-leads its strategic development of The Duchenne Registry, the largest patient-reported registry for people living with Duchenne muscular dystrophy.
Pat Furlong, MS, RN, BSN
Ms. Furlong is the founding president and CEO of Parent Project Muscular Dystrophy (PPMD) and one of the foremost authorities on Duchenne muscular dystrophy in the world. In 1994, she founded PPMD with other parents of young men who have Duchenne muscular dystrophy to change the course of the disease and ultimately find a cure. Together they accelerate research, raise their voices in Washington, D.C., demand optimal care, and educate the global community.
Emma James, PhD
Dr. James is vice president of medical affairs and patient advocacy at Encoded Therapeutics, a biotech company developing innovative gene therapies for patients with central nervous system disorders. She obtained her PhD from the University of Oxford, after completing her research at its Nuffield Department of Medicine and Wellcome Trust Centre for Human Genetics. With the majority of her career spent at small and mid-sized biotech companies in the United Kingdom, Boston, and San Francisco, Dr. James has focused the last seven years on developing gene therapies for rare, pediatric, and genetic disorders. In 2018, she was awarded Honorary Membership of the Faculty of Pharmaceutical Medicine of the United Kingdom's Royal College of Physicians in recognition of her contributions to pharmaceutical medicine.
Lisa Kearns, MS, MA
Ms. Kearns is a senior research associate in the Division of Medical Ethics at NYU Langone. For the past six years she has studied the ethical issues surrounding preapproval and expanded access to investigational drugs and state and federal right to try legislation. More recently, she has been studying the ethics of “bespoke” n-of-1 gene therapies. She is the deputy chair of the Compassionate Use Advisory Committees (CompAC), and participates in the NYU-University of Ghana Research Integrity Training Program.
Aisha Langford, PhD, MPH
Dr. Langford is an assistant professor in the Department of Population Health at NYU Langone and co-directs the Recruitment and Retention Core within NYU Langone Health’s Clinical and Translational Science Institute. She explores how health communication can improve individual decision-making and reduce population health disparities for conditions or behaviors that lead to preventable mortality and morbidity. Dr. Langford’s current research focuses on cardiovascular disease and cancer prevention and control.
Andrew McFadyen, BEd
Mr. McFadyen is executive director of the Isaac Foundation, a nonprofit organization he founded to fund research on mucopolysaccharidosis, a rare and progressive disease his elder son has. Andrew has led numerous advocacy efforts to shape public policy throughout Canada regarding the availability of treatments for children dying from rare diseases, and he fights for fair and equitable access to treatments for children who have rare diseases throughout Canada and the U.S.
Timothy M. Miller, MD
Dr. Miller is the head of PPD Inc’s Rare Disease & Pediatrics Center of Excellence and global vice president in development. Dr. Miller has spent the last 24 years focused exclusively in rare disease-related pursuits, both in the clinic and in the pharmaceutical industry, designed to reduce the unmet medical needs of patients and families affected by numerous rare and pediatric diseases. He currently works with a growing number of biopharmaceutical and biotech companies to identify and execute on their clinical development missions while advocating for greater patient centricity in research.
Patrick Moeschen, MA
Mr. Moeschen is a middle school music teacher. He was diagnosed with Becker muscular dystrophy (MD) in 1985 and re-diagnosed with limb-girdle MD, type 2E, in 2012. He earned a master’s in counseling and human relations from Liberty University in 2006, continues to teach full-time, and was awarded the Teacher of The Year for the state of New Hampshire in 2007. Patrick is an advocate for children and adults who have MD and has presented at disability conferences across the U.S., as well as in Australia, Italy, Belgium, and the Netherlands.
Erin Talati Paquette, MD, JD, MBe (Bioethics)
Dr. Paquette is an assistant professor in the Department of Pediatrics, Critical Care, at Northwestern University’s Feinberg School of Medicine, and a professor by courtesy at its Pritzker School of Law. She is also the chair of the Ethics Advisory Board and associate director of clinical and organizational ethics at the Ann & Robert H. Lurie Children’s Hospital of Chicago, a member of the steering committee for the Northwestern University Center for Bioethics, and the ethics representative on the Illinois Department of Public Health Genetic and Metabolic Diseases Committee. Dr. Paquette’s research considers issues of health equity, health disparities, and social justice. She has written and spoken extensively on informed consent for pediatric research, including the enrollment of children at risk for health disparities and enrollment of children into biorepositories.
Brendan Parent, JD
Professor Parent is an assistant professor in the Departments of Population Health and Surgery at NYU Langone and the director of transplant ethics and policy research in the Department of Population Health’s Division of Medical Ethics. He is also a fellow of the Center for Genetics and Society, a member of the Tri-SCI Embryonic Stem Cell Research Oversight Committee, and chair of the Ethical Issues in Health Care Committee of the New York State Bar Association.
Holly Tabor, PhD
Dr. Tabor is an associate professor of medicine and of health research and policy in epidemiology at the Stanford University Medical Center. She is a member of ethics committees for Stanford Hospital and Clinics and the Lucille Packard Children’s Hospital. She received her doctorate in epidemiology, with a minor in genetics, from Stanford University School of Medicine.