Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate

Neospora caninum is an obligate intracellular protozoan that affects several animal species. It is not pathogenic for humans, and its ability to infect and lyse a variety of cells and stimulate the immune system makes it an interesting drug candidate in oncology. The intrinsic oncolytic properties o...

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Bibliographic Details
Published in:Gene therapy 2024-05, Vol.31 (5-6), p.295-303
Main Authors: Guerriaud, Mathieu, Poupet, Cyril, Lakhrif, Zineb, Kohli, Evelyne, Moiré, Nathalie
Format: Article
Language:English
Subjects:
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Animals
Biomedical and Life Sciences
Biomedicine
Cancer therapies
Cell Biology
Cell therapy
Clinical trials
Drug development
Gene deletion
Gene Expression
Gene Therapy
Genetic engineering
Genetic Therapy - methods
Genomes
Human Genetics
Humanities and Social Sciences
Humans
Immune system
Law
Life Sciences
Nanotechnology
Neoplasms - genetics
Neoplasms - therapy
Neospora - genetics
Neospora - metabolism
Oncolysis
Protozoa
Safety
Suicide
Suicide genes
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Summary:Neospora caninum is an obligate intracellular protozoan that affects several animal species. It is not pathogenic for humans, and its ability to infect and lyse a variety of cells and stimulate the immune system makes it an interesting drug candidate in oncology. The intrinsic oncolytic properties of N. caninum have been confirmed in several preclinical models. Moreover, it can be modified to improve its safety and/or efficacy against cancer cells. In this study, we propose the legal categorization of this new biological drug candidate and the impact of modifications, notably the integration of a suicide gene, the deletion of a gene allowing its multiplication in healthy cells, and/or the insertion of a gene coding for a therapeutic protein into its genome. When unmodified, N. caninum can be categorized as a biological medicinal product, whereas modifications aimed at increasing its safety classify it as a Somatic Cell Therapy Medicinal Product, and modifications aiming to increase its efficacy or both safety and efficacy make it as a Gene Therapy Medicinal Product. This categorization is fundamental because it determines the guidelines applicable for preclinical development. These guidelines being numerous and complex, we have focused on the key requirements necessary for the development of the future medicinal product.
ISSN:0969-7128
1476-5462
DOI:10.1038/s41434-024-00445-1