Improving the assessment of risk factors relevant to potential carcinogenicity of gene therapies: a consensus article

Klapwijk, Jan C., Del Rio Espinola, Alberto, Libertini, Silvana, Collin, Philippe, Fellows, Mick D., Jobling, Susan, Lynch, Antohny M., Martus, HansJoerg, Vickers, Catherine, Zeller, Andreas, Biasco, Luca, Brugman, Martijn H., Bushmann, Frederic D., Cathomen, Toni, Ertl, Hildegund C. J., Gabriel, Richard, Gao, Guangping, Jadlowsky, Julie K., Kimber, Ian, Lanz, Thomas A., Levine, Bruce L., Micklethwaite, Kenneth P., Onodera, Masafumi, Pizzurro, Daniella M., Reed, Simon ORCID: https://orcid.org/0000-0002-4711-0560, Rothe, Michael, Sabatino, Denise, Salk, Jesse J., Schambach, Axel, Themis, Michael and Yuan, Jing 2024. Improving the assessment of risk factors relevant to potential carcinogenicity of gene therapies: a consensus article. Human Gene Therapy 35 (15-16) , pp. 527-542. 10.1089/hum.2024.033

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Abstract

Regulators and industry are actively seeking improvements and alternatives to current models and approaches to evaluate potential carcinogenicity of gene therapies (GTs). A meeting of invited experts was organized by NC3Rs/UKEMS (London, March 2023) to discuss this topic. This article describes the consensus reached among delegates on the definition of vector genotoxicity, sources of uncertainty, suitable toxicological endpoints for genotoxic assessment of GTs, and future research needs. The collected recommendations should inform the further development of regulatory guidelines for the nonclinical toxicological assessment of GT products.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Medicine
Publisher:	Mary Ann Liebert
ISSN:	1043-0342
Date of First Compliant Deposit:	16 August 2024
Date of Acceptance:	8 March 2024
Last Modified:	04 Sep 2024 14:19
URI:	https://orca.cardiff.ac.uk/id/eprint/171438

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