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2025 AABB Annual Meeting

Cord Blood

Emerging Ethics and Policy Issues in Cord Blood Therapeutics and Gene Therapies (President’s Session)

AM25-SN-01-L - CBC: Emerging Issues in Cord Blood Therapeutics and Gene Therapies (President’s Session)

Sunday, October 26, 2025

8:00 AM - 9:15 AM PT

Room: SDCC 32

CE: 0 CME (AMA Physician Credit) / 1.25 CE (Non-Physician Credit)

Program Chair(s)

Joanne Kurtzberg, MD

Jerome Harris Distinguished Professor of Pediatrics Professor of Pathology Director, Marcus Center for Cellular Cures Director, Carolinas Cord Blood Bank Co-Director, Stem Cell Transplant Laboratory President, Cord Blood Association
Duke University
Durham, North Carolina

Disclosure(s): Celularity: Consultant/Advisory Board (Ongoing); CryoCell International: Full-time/Part-time Employee or Owner (Ongoing); HemxBio: Consultant/Advisory Board (Ongoing); Mesoblast: Consultant/Advisory Board (Ongoing); Supercell (formally Sinocell): Duke license with Sinocell, received royalties (Ongoing)

Handouts

Speaker(s)

Jeremy Sugarman, MD, MPH, MA

Harvey M. Meyerhoff Professor of Bioethics & Medicine
Johns Hopkins University
Baltimore, Maryland

Disclosure(s): Aspen Neurosciences, Inc: Consultant/Advisory Board (Ongoing); Merck: Consultant/Advisory Board (Ongoing); Merck KGaA: Consultant/Advisory Board (Ongoing)
Peter W. Marks, MD, PhD (he/him/his)

Independent Consultant
Pharmaceutical Industry
Washington, District of Columbia

Disclosure(s): Independent Consultant: Consultant/Advisory Board (Ongoing)

This session will focus on the challenges to developing and bringing innovative cell and gene therapies to patients in need. Many of these initiatives involve patients with rare or ultra rare diseases where current models for ‘drug’ development do not apply. Furthermore, traditional business models for drug development are also a poor fit due to high complexity of manufacturing and small markets for product utilization leading to abandonment of patients who could greatly benefit from these therapies. Changes in disease modeling, product manufacturing, clinical trial design and translation, and requirements for commercialization will be discussed with the goal of redesigning approaches to commercialize promising cell and gene therapies.

Learning Objectives:

Examine the unique challenges associated with developing and delivering innovative cell and gene therapies for rare and ultra-rare diseases, including limitations of current drug development frameworks.
Identify gaps in traditional business models that hinder the commercialization of complex therapies with small market potential, and explore alternative approaches that prioritize patient access
Foster collaborative dialogue on how to redesign commercialization strategies to better serve patients who may otherwise be left behind by conventional development models.