As the Duchenne community enters the era of gene therapy, many new concepts, terms and issues arise, including those related to research design, patient autonomy, sibling and family involvement, fairness in access and the quality of information patients and families receive.

As part of PPMD’s Gene Therapy Initiative, we reached out to NYU’s Division of Medical Ethics with the goal of initiating a Working Group to identify and address these emerging ethical issues surrounding investigational gene therapy studies in Duchenne and the broader pediatric patient community.

The goal of the Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group, supported by funding from PPMD, is to advance research, policy, and education, by promoting improved understanding of the challenges and best practices for ethical research across the evolving landscape of genetic therapies. This working group includes a range of stakeholders to include patient advocacy, law, medicine, clinical research, and the biopharmaceutical industry.

WATCH: PGTME’s Educational Lecture Series

The PGTME Working Group recently hosted a lecture series on a range of topics relevant to our community, including the FDA perspective, AAV serotypes, acquired and innate immunity, informed consent and the patient perspective. It is our hope that these lectures will provide the community with educational tools as we move through development of potential gene therapies for Duchenne.

All talks can be accessed by the community and watched below to help supplement the learnings PPMD has previously shared and continue to build everyone’s base of knowledge. We also recognize that some of the concepts presented can be difficult to grasp, so we will continue to share additional resources where families can learn about gene therapy.

Equity Issues in Pediatric Gene Therapy Research

Talk by Dr. Pilar Ossorio, Professor of Law and Bioethics at University of Wisconsin Law School, with a response by Rafael Escandon, Senior Vice President of Medical Affairs, Policy, and Patient Engagement at BridgeBio Pharma Inc.

Moderated by Dr. Aisha Langford, Assistant Professor, Department of Population Health, NYU Grossman School of Medicine.

Ethical Challenges of Immunogenicity & Toxicity in Pediatric Gene Therapy Research

Talk by Dr. James Wilson, Director of UPenn Perelman School of Medicine’s Gene Therapy program, with a response by Dr. Timothy Cripe, Chief of Pediatric Hematology and Oncology and Blood and Marrow Transplantation at Nationwide Children’s Hospital.

Moderated by Katherine Beaverson, MS, Patient Advocacy lead for the Rare Disease Research Unit (RDRU) at Pfizer Inc.

Lived Experiences The Personal + Family Impact of Participating in Pediatric Gene Therapy Research

Talk by John F. Crowley, Chairman and CEO of Amicus Therapeutics, with a response by Patrick Moeschen, Muscular Dystrophy patient advocate.

Moderated by Dr. Moshe Cohn, clinical assistant professor in the Department of Pediatrics at NYU Langone.

Risks & Benefits in Pediatric Gene Therapy Research

Talk by Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, with a response by Pat Furlong, founder and president of Parent Project Muscular Dystrophy.

Moderated by Dr. Alison Bateman-House, PhD, MPH, MA, PGTME Co-Chair

Operationalizing Informed Consent in Pediatric Gene Therapy Research

Talk by Sandy Macrae, CEO & President of Sangamo Therapeutics, Inc., with a response by Dr. Art Caplan, Mitty Professor of Bioethics, NYU Grossman School of Medicine, Founding Director of the NYU Grossman Division of Medical Ethics

Moderated by Dr. Lesha Shah, PGTME Co-Chair and assistant professor of psychiatry and the medical director of Child, Adolescent and Family Services at the Icahn School of Medicine at Mount Sinai