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How to pay for individualized genetic medicines

For precision genetic medicines to fulfill their potential as treatments for ultra-rare diseases, fresh approaches to academic–industry partnerships and data sharing are needed, together with regulatory change and adaptation of reimbursement models.

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Fig. 1: The comparable costs of transplantation and gene therapy.

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Acknowledgements

The authors thank partners at the N=1 Collaborative, the Yu laboratory and EveryONE Medicines for contributions and collaboration. We thank J. Cummings for editorial assistance.

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Correspondence to Julia M. Y. Pian or Timothy W. Yu.

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Competing interests

J.M.Y.P. is an associate at Atlas Venture. J.V. is a co-founder of EveryONE Medicines and the N=1 Collaborative. W.X.Y. is a founder, employee and shareholder of Arbor Biotechnologies. A.W.L. reports personal investments in private biotechnology companies, biotechnology venture capital funds and mutual funds; is a co-founder and principal of QLS Advisors LLC, a healthcare investments advisor, and QLS Technologies LLC, a healthcare analytics and consulting company; a director of AbCellera, Annual Reviews, Atomwise, BridgeBio Pharma, Uncommon Cures and Vesalius Therapeutics; an advisor to Apricity Health, Aracari Bio, BrightEdge Impact Fund, Enable Medicine, FINRA, Health at Scale, MIT Proto Ventures, Quantile Health, Roivant Social Ventures, Swiss Finance Institute, Thalēs, Think Therapeutics and xCures; and during the most recent 6-year period has received speaking/consulting fees, honoraria, or other forms of compensation from AbCellera, AlphaSimplex, Annual Reviews, Apricity Health, Aracari Bio, Atomwise, Bernstein Fabozzi Jacobs Levy Award, BridgeBio, Cambridge Associates, CME, Enable Medicine, Journal of Investment Management, Lazard, MIT, New Frontier Advisors, Oppenheimer, Princeton University Press, Q Group, QLS Advisors, Quantile Health, Research Affiliates, Roivant, SalioGen Therapeutics, Swiss Finance Institute, Think Therapeutics, Vesalius Therapeutics and WW Norton. T.W.Y. has received research funding from EveryOne Medicines, has served as a scientific consultant to Biomarin and Servier Pharmaceuticals, is a board member of the Oligonucleotide Therapeutics Society and serves as a volunteer scientific advisor to several nonprofit rare disease foundations. J.M.Y.P., J.V., T.W.Y. and W.X.Y. are volunteers with the N=1 Collaborative, a non-profit organization.

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Pian, J.M.Y., Owusu, N., Vitarello, J. et al. How to pay for individualized genetic medicines. Nat Med 30, 1816–1818 (2024). https://doi.org/10.1038/s41591-024-03071-x

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