Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence
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biomedicines-13-00758-v2.pdf
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1.63 MB
Format
Adobe PDF
Checksum (MD5)
f28ff03fc617061f1c6a9c9479f1a89a
Author(s)
Olaghere, Jimi
Williams, David A.
Farrar, Jeremy
Büning, Hildegard
Calhoun, Cecelia
Ho, Tony
Inamdar, Maneesha S.
Liu, David
Makani, Julie
Nyarko, Kwasi
Date Issued
March 20, 2025
Journal
Biomedicines
Publisher
Multidisciplinary Digital Publishing Institute
Citation
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.
Version
Final published version
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.
MIT Department
Broad Institute of MIT and Harvard
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DOI of Published Version
https://doi.org/10.3390/biomedicines13030758
