Platform-based approaches for gene-editing therapies could markedly improve development efficiency, reduce costs and increase access for patients with rare diseases. Although gene editing has shown remarkable clinical success for a small number of Mendelian disease indications, broader adoption faces substantial hurdles. We propose strategies to overcome these challenges through modular platforms for nonclinical and chemistry, manufacturing and controls (CMC) data reuse, risk-based manufacturing quality, and streamlined umbrella clinical trials for regulatory efficiency and accelerated approval.
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References
Frangoul, H. et al. N. Engl. J. Med. 384, 252–260 (2021).
Gillmore, J. D. et al. N. Engl. J. Med. 385, 493–502 (2021).
Mast, J. The CRISPR companies are not OK: how hype, scientific setbacks, and growing investor demands humbled the gene editing industry. STAT+ https://www.statnews.com/2025/02/06/crispr-gene-editing-medical-breakthrough-not-matched-by-financial-success/ (2025).
Urnov, F. et al. Cytotherapy https://doi.org/10.1016/j.jcyt.2025.06.010 (2025).
Frangoul, H. et al. N. Engl. J. Med. 390, 1649–1662 (2024).
Verve Therapeutics. Verve Therapeutics announces pipeline progress and anticipated 2025 milestones. vervetx.com https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-pipeline-progress-and-anticipated (2025).
Tice, J. A. et al. Gene Therapy for Hemophilia B and An Update on Gene Therapy for Hemophilia A: Effectiveness and Value; Evidence Report (Institute for Clinical and Economic Review, 2022).
National Academies of Sciences, Engineering, and Medicine. Emerging Technologies and Innovation in Manufacturing Regenerative Medicine Therapies: Proceedings of a Workshop –in Brief (The National Academies Press, 2024).
National Academies of Sciences, Engineering, and Medicine. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop (The National Academies Press, 2020).
Mackall, C. L. et al. Nat. Med. 30, 1836–1846 (2024).
Ecker, D. J. et al. Nat. Rev. Drug Discov. 23, 321–322 (2024).
Acknowledgements
The authors thank FDA participants for their active and substantial engagement during the scientific exchange. The exchange was supported by the Alliance for Regenerative Medicine, the International Society for Cell & Gene Therapy and Danaher Corporation.
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S.H.K. is an employee and holds equity in Danaher and is an advisor to nChroma Bio. F.U. is an advisor and holds equity in Tune Therapeutics. K.M. is an advisor to and holds equity in Verve Therapeutics and Variant Bio, is an advisor to LEXEO Therapeutics and Capstan Therapeutics, and receives research funding from Nava Therapeutics and Beam Therapeutics. A. Lee is an employee and holds equity in Prime Medicine. L.B. is an employee and holds equity in Beam Therapeutics. P.S. is an employee and holds equity in Arbor Biotechnologies. J.B. is an employee and holds equity in CRISPR Therapeutics. M.H. is an employee and holds equity in Charles River Labs. T.L. is an employee and holds equity in Verve Therapeutics. V.A.-N. is an employee and holds equity in Danaher. H.M. participated as an expert in the field, and opinions expressed by him at the meeting or in this commentary do not represent National Institutes of Health policy or opinion. D.R.L. is a co-founder, consultant, and/or equity holder of Beam Therapeutics, Prime Medicine, Pairwise Plants, nChroma Bio, and Exo Therapeutics.
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Kassim, S.H., Urnov, F., Musunuru, K. et al. Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable. Nat Biotechnol 43, 1047–1049 (2025). https://doi.org/10.1038/s41587-025-02744-3
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DOI: https://doi.org/10.1038/s41587-025-02744-3
