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Therapeutic genome editing: regulatory horizons

An increasing number of genome-editing technologies are being developed. Here, we highlight some of the key issues for future therapies using these technologies from a regulatory perspective.
  1. Philip A. Hines

    1. European Medicines Agency, Amsterdam, Netherlands; The United Nations University–Maastricht Economic and Social Research Institute on Innovation and Technology, Maastricht, Netherlands.

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  2. Eleonora Agricola

    1. Italian Medicines Agency, Rome, Italy.

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  3. Jordi Llinares Garcia

    1. European Medicines Agency, Amsterdam, Netherlands. *Deceased.

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  4. Laurence O’Dwyer

    1. Health Products Regulatory Authority (HPRA), Dublin, Ireland.

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  5. Ralf Herold

    1. European Medicines Agency, Amsterdam, Netherlands.

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Genome-editing agents have the potential to treat and perhaps even cure a growing range of diseases with a single dose1, which contrasts with the chronic dosing regimens often needed with other medicines. Various tools continue to be developed for genome editing, including zinc finger nucleases, transcription activator-like effector nucleases (TALENs), CRISPR–Cas9 editors and prime editors, which can be used to repair, modulate, replace or add genes to achieve a desired genotype. Although such tools are far from perfect, with issues such as off-target editing and delivery challenges needing to be addressed, ongoing advances are making genome editing more precise, more efficient and less expensive.

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Nature Reviews Drug Discovery 21, 1-2 (2022)

doi: https://doi.org/10.1038/d41573-021-00130-7

Acknowledgement

This article is dedicated to the memory of Jordi Llinares Garcia, a life committed to public health.

Disclaimer

The views expressed in this article are the personal views of the author(s) and may not be understood or quoted as being made on behalf of or reflecting the position of the regulatory agency/agencies or organizations with which the author(s) is/are employed/affiliated.

References

  1. Mullard, A. Gene-editing pipeline takes off. Nat. Rev. Drug Discov. 19, 367–372 (2020).

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  2. van Haasteren, J. et al. The delivery challenge: fulfilling the promise of therapeutic genome editing. Nat. Biotechnol. 38, 845–855 (2020).

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  3. Teboul, L. et al. Variability in genome editing outcomes: challenges for research reproducibility and clinical safety. Mol. Ther. 28, 1422–1431 (2020).

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  4. World Health Organization. Human Genome Editing. As we explore options for global governance, caution must be our watchword. WHO.int https://www.who.int/ethics/topics/human-genome-editing/ethics-explore-options-for-global-governance.pdf?ua=1 (2019).

  5. National Academy of Medicine, National Academy of Sciences & The Royal Society. Heritable Human Genome Editing https://doi.org/10.17226/25665 (The National Academies Press, 2020).

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Competing Interests

The authors declare no competing interests.

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