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.
Alen Agaronov, ScD
Dr. Agaronov is a postdoctoral fellow in the Division of Medical Ethics. A member of PGTME's sister working group on Compassionate Use and Preapproval Access, and the latter's subgroup on Ethics and Real-World Evidence, he studies how industries, regulators, payers, providers, and patient advocacy organizations tend to co-construct and will into being the clinical evidence backing promising therapies for rare and infectious diseases. Crossing the practicality of public science with the intellectual pursuit of science and technologies studies, he is currently partnering with stakeholders to develop and make sense of new clinical outcomes assessments for new medical technologies that target unmet needs. Dr. Agaronov received an ScD in social and behavioral sciences from the Harvard T.H. Chan School of Public Health with a secondary field in critical media practice through The Film Study Center at Harvard University, and a BS-MS dual degree from CUNY Hunter College. Contact him at email@example.com.
Rasha Alnaibari, MBA
Ms. Alnaibari started her career in asset management on the buy side. After obtaining her MBA from INSEAD, she joined Shell Gas and Power where she helped lead Shell LNG Trading's Middle East division. When her first child was born, she and her husband moved back to Kuwait where she built a global portfolio of infrastructure and energy investments for Kuwait Fund. In 2016, her son was diagnosed with Duchenne muscular dystrophy. She is now based in New York, where her husband is a diplomat and her boys go to school.
Marilyn Baffoe-Bonnie, MA
Marilyn Baffoe-Bonnie is a PhD candidate in the Department of Sociology at Rutgers University. She holds a predoctoral fellow appointment in the Department of Bioethics at the National Institutes of Health and the Health Disparities Unit of the National Human Genome Research Institute's Social and Behavioral Science Research Branch. Her work utilizes mixed methods to examine the ethical, legal, social, and cultural implications of emerging biotechnologies with a focus on uncovering how they may reproduce health inequities. Her current research focuses on the ethical and social dimensions of using gene therapy and gene editing to treat sickle cell disease.
Katherine Beaverson, MS
Ms. Beaverson is a global rare disease advocacy lead at Pfizer Inc., guiding the strategic planning and implementation of collaborations with patient advocacy groups to integrate patient expertise into early medicines development and delivery continuum. 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 M. Cohn, MD, HEC-C
Dr. Cohn is affiliated faculty in the Division of Medical Ethics in NYU Langone’s Department of Population Health. He practices pediatric critical care and hospice and palliative medicine, and is a certified healthcare ethics consultant. 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 currently a consultant to the biotechnology industry and most recently was the senior vice president of medical affairs, policy, and patient engagement at BridgeBio Gene Therapy. He has worked in global clinical research and development and patient advocacy in the biotechnology industry for nearly 30 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 clinical and medical affairs and head of medical office at Encoded Therapeutics, a biotech company developing gene therapies for children 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 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 spent the past decade focused on developing gene therapies for rare pediatric 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. She has studied the ethical issues surrounding preapproval to investigational drugs and more recently the ethics of individualized gene therapies. She is a member of the division's Working Group on Compassionate Use and Preapproval Access and its subgroup Bioethics of Individualized Therapeutics. She is deputy chair of the Compassionate Use Advisory Committees and associate director of the division’s High School Bioethics Project.
John D. Lantos, MD
Dr. Lantos is a bioethicist and primary care pediatrician in New York City. He was formerly a professor of pediatrics at both the University of Chicago and the University of Missouri. His research focuses on innovative treatment for children, and he has written about ethical issues in neonatology, oncology, endocrinology, and genomics. He was a co-principal investigator of a National Institutes of Health (NIH)–sponsored study of Newborn Sequencing in Genomic Medicine and Public Health and a Patient-Centered Outcomes Research Institute (PCORI)–funded study of community-driven participatory research in genomics and rare disease. From 2013 to 2016, he served as co-chair of the PCORI Clinical Trials Advisory Panel and was an advisor to the American Academy of Pediatrics primary care research network. He is past president of the American Society for Bioethics and Humanities and the American Society of Law, Medicine, and Ethics. His most recent book, Kidney to Share, examines ethical issues in living kidney donation.
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 United States.
Timothy M. Miller, MD
Dr. Miller leads the Rare Disease and Pediatrics work of PPD, Inc., part of Thermo Fisher Scientific, a global research organization that is focused on delivering transformational therapies to patients and families affected by rare diseases. He currently serves as vice president and global therapeutic area head, leading cross-functional teams in the delivery of novel research programming in a variety of indications and across phases. Dr. Miller has spent the past 25 years focused on rare disease–related care and research pursuits, both in the clinic and in the pharmaceutical industry, with an emphasis on the reduction of unmet medical need for patients and families. He currently works with 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 recently retired after 28 years of teaching music, instruments, jazz, and concert bands at the middle school level. Diagnosed with Becker muscular dystrophy (MD) in 1985 and re-diagnosed with limb-girdle MD type 2E in 2012, Patrick continues to lead an active life working in education and MD advocacy. Since 2002, Patrick has been working with Parent Project Muscular Dystrophy (PPMD), running the 40-member Patient Adult Advisory Committee (PAAC). The PAAC elevates the patient voice in lobbying Congress, as well as organizing, designing, and implementing workshops, webinars, and seminars at disability conferences throughout the world. Patrick is also involved with PPMD in the area of female manifesting carriers of Duchenne muscular dystrophy. This underserved population is a growing voice in the rare disease arena, and Patrick has organized a group that is working with an adult clinic at the University of Pennsylvania with a goal of educating the medical community about the challenges that these women face.
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 an associate 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.