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 Division of Medical Ethics, Department of Population Health, at NYU Grossman School of Medicine. At the division, in addition to PGTME, she co-chairs the 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 Johnson & Johnson on the ethical allocation of its investigational products. In 2023, she was appointed a member of the US Department of Health and Human Services Secretary’s Advisory Committee on Human Research Protections (SACHRP). 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.

Ayden Eilmus, Program Coordinator

Ms. Eilmus’s research background is in the ethical, legal, and social implications (ELSI) of genetics and genomics, and the portrayal of emerging technology and bioethics in popular culture. She received her BA from Vanderbilt University in 2021, where she triple majored in anthropology, philosophy, and medical humanities.

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 postdoctoral fellow in the Department of History and Sociology of Science at the University of Pennsylvania. Her work utilizes quantitative, qualitative, and computational 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 concerns the ethical and social dimensions of gene therapy for sickle cell disease with a focus on lived experience and health communication. Dr. Baffoe-Bonnie earned a PhD in sociology from Rutgers University. She completed her predoctoral fellowship at the National Institutes of Health Clinical Center Department of Bioethics and the National Human Genome Research Institute’s Health Disparities Unit in the Social and Behavioral Research Branch. She completed her master’s of bioethics at the University of Pennsylvania and her BS in psychology from Haverford College.

Katherine Beaverson, MS

Ms. Beaverson is a vice president of global patient advocacy at Dyne Therapeutics, guiding the strategic planning and implementation of collaborations with patient advocacy groups to integrate patient expertise into early medicines development and delivery continuum. She held similar roles in industry with Pfizer, Inc., Boehringer Ingelheim and Amicus Therapeutics. 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.

Allyson Berent, DVM

Dr. Berent is a veterinary internal medicine specialist and the director of Interventional Endoscopy Services at the largest animal teaching hospital in the world, the Animal Medical Center, in New York City. In 2014 her daughter was diagnosed with Angelman syndrome, a rare nondegenerative neurogenetic disorder. In October 2015 she joined the board of directors for the Foundation for Angelman Syndrome Therapeutics (FAST) and became chief science officer for the Foundation. She helped spearhead the development of a pre-competitive biomarker and outcome measure consortium to bring patient-focused outcome measures forward for human clinical trials and now serves as the co-director of this consortium. Currently she is working to advance numerous cell and gene therapy products through IND-enabling studies into first in human clinical trials for Angelman syndrome through her efforts at FAST and its venture philanthropy model; she also serves as the chief development officer of its newly founded drug accelerator, called AS2 Biotherapeutics.

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.

Carolyn Riley Chapman, PhD, MS

Dr. Chapman joined the Multi-Regional Clinical Trials Center of Brigham and Women's Hospital and Harvard (MRCT Center) in October 2023. She is a lead investigator in the Division of Global Health Equity (DGHE), Department of Medicine, Brigham and Women's Hospital and a member of the faculty of the Department of Medicine at Harvard Medical School. Her work involves collaboration with diverse stakeholders to identify and address challenges in the research and development of precision medicine, including cell and gene therapies. Prior to joining the MRCT Center, Dr. Chapman worked at NYU Grossman School of Medicine in various academic roles. In the past, she has worked for the Columbia Bioethics program, L.E.K. Consulting, and Aton Pharma, and as a freelance science/medical writer. She has a PhD in genetics from Harvard and an MS in bioethics from Columbia.

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 170 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 principal and founder of DGBI Clinical Research and Ethics Consulting on Bainbridge Island, Washington. 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 section faculty at the Mercy Children’s Bioethics Center in Kansas City and 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. 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.

Ryan Fischer

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, MFPM (Hon)

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 more than a decade specifically 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.

Liza-Marie Johnson, MD, MPH, MSB, HEC-C

Dr. Liza-Marie Johnson is an associate member (professor) in the Department of Oncology and the director of the Bioethics Program at St. Jude Children’s Research Hospital. She is board-certified in pediatrics and pediatric hematology-oncology. Her academic work focuses on clinical and research ethics, particularly concerning pediatric populations. Dr. Johnson collaborates with patients and families to enhance patient-provider communication and improve the quality of the informed consent process, especially for novel or high-risk interventions, ensuring it meets their informational needs. Currently, she is developing an educational toolkit about treatment options for sickle cell disease, with an emphasis on gene therapy. On a national level, Dr. Johnson serves as the bioethicist for two NIH data safety monitoring boards. She co-chairs the Bioethics Special Interest Group of the American Pediatric Association, is the immediate past chair of the bioethics committee for the Children’s Oncology Group, and is a member of the steering committee of the Clinical Research Ethics Consultation Collaborative.

Lisa Kearns, MS, MA

Ms. Kearns is an associate research scientist 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. In addition to PGTME, she is a member of the division's Working Group on Compassionate Use and Preapproval Access. She is deputy chair of the Compassionate Use Advisory Committees, independent panels of clinicians, ethicists, and patient representatives that advise Janssen Pharmaceuticals on the ethical allocation of its investigational medical products. She is also associate director of the division's High School Bioethics Project.

Laura L. Kimberly, PhD, MSW, MBE

Dr. Kimberly is an assistant professor in the Hansjörg Wyss Department of Plastic Surgery and in the Division of Medical Ethics in the Department of Population Health at NYU Grossman School of Medicine. Her current research examines ethical and psychosocial implications of innovative medical and surgical interventions, with a focus on health equity and quality of life across the lifespan. She employs qualitative and mixed methods approaches to center patients' lived experiences of health care in content areas including organ transplantation, gender-affirming care, and pediatric gene therapy clinical trials. Dr. Kimberly holds a PhD in social work from Columbia University and master's degrees in bioethics and social work from the University of Pennsylvania.

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

Andrew McFadyen is the ethics lead for Precision Child Health at the Hospital for Sick Children in Toronto, Ontario, Canada. Before that, he was the executive director of two patient advocacy organizations for patients and families with rare diseases. He has worked closely with governments and pharmaceutical companies to help ensure access to novel therapeutics for patients in need, as well as how to create equitable expanded access programs. Mr. McFadyen has testified as an expert witness in the U.S. Senate and Canadian parliamentary steering committees on access to treatments for patients with rare diseases. He is a longtime member of NYU’s Division of Medical Ethics Working Group on Compassionate Use & Preapproval Access (CUPA). His interest in rare diseases, novel therapeutics, and access to medicines began when his son Isaac was diagnosed with Mucopolysaccharidosis VI (MPS VI). Mr. McFadyen spent 15 years as an elementary school teacher, and he holds a BA and BEd from Queen’s University and an MHSc in bioethics from the University of Toronto. He has an academic appointment at the Dalla Lana School of Public Health (DLSPH) at the University of Toronto as an adjunct lecturer.

Timothy M. Miller, MD

Dr. Miller is a patient advocate, biochemist and former academic neurologist who currently leads the Enterprise Science & Innovation team at Thermo Fisher Scientific and formerly led the Rare Disease & Pediatrics research at PPD, Inc, both global organizations focused on serving science and delivering transformational therapies to patients and families. He is the co-chair of the Accelerating Medicines Partnership/Foundation of the NIH's Bespoke Gene Therapy Consortium, a monumental public/private partnership leading the development of multiple first in human AAV gene therapy studies in rare disease indications with the goal of advancing our systems involved in the delivery of the promise of these exciting therapies. In his day job, Dr. Miller leads cross-functional teams seeking to drive scientific and in-silico innovation with both large and small therapeutic development entities. He has spent the past 25 years focused on advancing the state of therapeutics for patients affected by rare diseases, both in academic clinics and the pharmaceutical industry.

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 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.