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Volume 13
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Conference Report

Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence

by
Jimi Olaghere
1,
David A. Williams
2,3,
Jeremy Farrar
4,
Hildegard Büning
5,
Cecelia Calhoun
6,
Tony Ho
7,
Maneesha S. Inamdar
8,
David Liu
9,
Julie Makani
10,
Kwasi Nyarko
11,
Sol Ruiz
12,
John Tisdale
13,
Joseph M. McCune
14,
Esther Boadi
15,* and
Reagan-Udall Foundation for the FDA
1
Sugarloaf Capital, Atlanta, GA 30309, USA
2
Boston Children’s Hospital, Boston, MA 02115, USA
3
Harvard Medical School, Boston, MA 02115, USA
4
World Health Organization, Avenue Appia 20, 1211 Genève, Switzerland
5
Hannover Medical School, 30625 Hannover, Germany
6
School of Medicine, Yale University, Orange, CT 06477, USA
7
Pivotal Lifesciences, Cambridge, MA 02142, USA
8
Institute for Stem Cell Science and Regenerative Medicine, Bangalore 560065, India
9
Broad Institute of Massachusetts Institute of Technology (MIT) and Harvard, Cambridge, MA 02142, USA
10
Department of Haematology and Blood Transfusion, Muhimbili University of Health and Allied Sciences (Tanzania), Dar-Es-Salaam P.O. Box 65001, Tanzania
11
WHO Regional Office for Africa (WHO-AFRO), Cité du Djoué, Brazzaville P.O. Box 06, Congo
12
Spanish Medicines Agency (AEMPS), C/Campezo, 1, Edificio 8, 28022 Madrid, Spain
13
National Heart, Lung, and Blood Institute (NIH), Bethesda, MD 20892, USA
14
Gates Foundation, Seattle, WA 98109, USA
15
Reagan-Udall Foundation for the FDA, Washington, DC 20036, USA
 
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*
Author to whom correspondence should be addressed.
Biomedicines 2025, 13(3), 758; https://doi.org/10.3390/biomedicines13030758
Submission received: 31 January 2025 / Revised: 22 February 2025 / Accepted: 18 March 2025 / Published: 20 March 2025
(This article belongs to the Section Gene and Cell Therapy)
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Abstract

On 4 September 2024, the Reagan-Udall Foundation for the FDA (FDA Foundation) in collaboration with the Food and Drug Administration (FDA) and the Gates Foundation hosted a workshop titled “Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence”. The event brought together a diverse group of experts, including international regulatory bodies, regulated industries, healthcare professionals, patients, academic researchers and global health advocates, to discuss the rapid advancements in gene therapy and the pressing need for equitable access in low-and middle-income countries (LMICs), with sickle cell disease (SCD) serving as the model disorder for the discussions. Although there has been significant progress in gene therapy, such as breakthroughs in clustered regularly interspaced short palindromic repeats (CRISPR)-based technologies and FDA-approved therapies, access to these therapies remain limited in underresourced regions. The workshop addressed critical challenges, including the high cost of therapies, regulatory gaps and barriers and ethical concerns regarding informed consent and public engagement in LMICs. This paper highlights the critical discussion points from the workshop with a focus on exploring strategies for global regulatory convergence, the role of international collaborations and the potential pathways to making gene therapies affordable and accessible to all.
Keywords: gene therapy; low-and middle-income countries (LMICs); sickle cell disease (SCD); clustered regularly interspaced short palindromic repeats (CRISPR); chimeric antigen receptor T cell (CAR T cell) gene therapy; low-and middle-income countries (LMICs); sickle cell disease (SCD); clustered regularly interspaced short palindromic repeats (CRISPR); chimeric antigen receptor T cell (CAR T cell)

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MDPI and ACS Style

Olaghere, J.; Williams, D.A.; Farrar, J.; Büning, H.; Calhoun, C.; Ho, T.; Inamdar, M.S.; Liu, D.; Makani, J.; Nyarko, K.; et al. Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence. Biomedicines 2025, 13, 758. https://doi.org/10.3390/biomedicines13030758

AMA Style

Olaghere J, Williams DA, Farrar J, Büning H, Calhoun C, Ho T, Inamdar MS, Liu D, Makani J, Nyarko K, et al. Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence. Biomedicines. 2025; 13(3):758. https://doi.org/10.3390/biomedicines13030758

Chicago/Turabian Style

Olaghere, Jimi, David A. Williams, Jeremy Farrar, Hildegard Büning, Cecelia Calhoun, Tony Ho, Maneesha S. Inamdar, David Liu, Julie Makani, Kwasi Nyarko, and et al. 2025. "Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence" Biomedicines 13, no. 3: 758. https://doi.org/10.3390/biomedicines13030758

APA Style

Olaghere, J., Williams, D. A., Farrar, J., Büning, H., Calhoun, C., Ho, T., Inamdar, M. S., Liu, D., Makani, J., Nyarko, K., Ruiz, S., Tisdale, J., McCune, J. M., Boadi, E., & Reagan-Udall Foundation for the FDA. (2025). Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence. Biomedicines, 13(3), 758. https://doi.org/10.3390/biomedicines13030758

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