Since the first gene therapy was approved in 2017, more than 30 such therapies have been approved worldwide. The U.S. Food and Drug Administration (FDA) expects this number to grow significantly, as more than 2,000 gene therapies are currently in development.

To start the day’s scientific program on Feb. 15, a panel of experts will address the accessibility of gene therapy around the world in the session Welcome, Awards & Plenary: Gene Therapy. The discussion will begin at 8:15 a.m. in Ballroom B.

Session Chair Jennifer E. Adair, PhD, FUNAS, associate professor at the Fred Hutchinson Cancer Center and associate research professor at the University of Washington, will discuss the current state of gene therapy. Most gene therapies are authorized for diseases that represent global health burdens, including cancer, hemophilia, and sickle cell disease. While these are approved in the United States and Europe, most patients diagnosed with these diseases are in lower-middle-income countries, as defined by the World Bank.

“This means that we have something that’s working effectively in a disease that represents a global health burden, but all of the testing is being done in places that absolutely don’t represent the majority of patients worldwide,” Adair said.

By restructuring the design and development of gene therapies, Adair contends that access to clinical trials and therapy can be increased in the settings with the most patients.

“Thinking about socially responsible development of these therapies and access to them requires thought from the beginning of whatever your gatekeeping interaction is with that patient pool,” she said. “For researchers, it means designing the studies from the beginning to think about all possible patients. In a clinical trial, it means thinking not just about the patients that you see in your clinic but of all the patients in the world that are impacted by this disease.”

Another panelist, Claire Booth, MBBS, PhD, professor of gene therapy and pediatric immunology at the University College of London and Great Ormond Street Hospital, London, United Kingdom, will discuss strategies to improve access to gene therapies for rare diseases.

Despite authorization, gene therapies for rare diseases are being withdrawn from the market due to low market values. The need to ensure access to these therapies is driving a new era of regulatory approvals. In this new era, a hospital might provide an authorized gene therapy for a rare disease at a low cost and not for profit.

Booth will discuss questions surrounding how academic institutions could afford to maintain authorization and when these therapies would enter back into the commercial phase.

Adair and Booth will be joined by Daniel Bauer, MD, PhD, who will discuss the science of gene therapy for hemoglobinopathies and the practical applications of this therapy, especially in patient populations with diverse genomes. He is a physician-scientist and director of the Gene Therapy Program at Dana Farber/Boston Children’s Cancer and Blood Disorders Center, and the Fredrickson Associate Professor of Pediatrics at Harvard Medical School.

This and other sessions at the 2025 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR® will be available for on-demand viewing for registered attendees following the live presentation.