MTE #5: Gene-Based Therapy in Liver Disease—Promise, Ethics, and Responsibilities (Ticketed)

Gene therapy trials for liver diseases present unprecedented opportunities for both patients and investigators. While offering the potential for durable disease modification for patients, gene therapies also raise complex questions for investigators regarding informed consent, heightened safety monitoring requirements, equity of access, pediatric enrollment, long-term follow-up obligations, and investigator responsibility for therapies with potentially irreversible effects. The Clinical Research Committee and Pediatric Special Interest Group jointly sponsor this expert session that provides an informal, small-group forum for in-depth discussion with recognized leaders in pediatric hepatology, genetics, and research ethics. Presenters focus on real-world challenges faced by investigators conducting liver-directed gene therapy trials for conditions such as urea cycle disorders, Wilson disease, and alpha-1 antitrypsin deficiency as well as acquired conditions such as hepatitis B. Discussion topics include: managing uncertainty and expectations; balancing innovation with patient protection; long-term follow-up obligations; and how investigators can responsibly integrate gene therapy into clinical research and practice. Speakers aim to equip attendees with the tools necessary to ensure safe, ethical, and effective implementation of these trials across research sites.