改善对基因疗法的潜在致癌性相关的风险因素的评估:一个共识文章
Jan C Klapwijk1, Alberto Del Rio Espinola2, Silvana Libertini3
1Cornelis Consulting Ltd, London, United Kingdom.
Human gene therapy
|July 25, 2024
概括
专家们召开了会议,以定义载体基因毒性,并确定基因疗法 (GTs) 适合的毒理终点. 这些建议旨在改善致癌性评估,并指导非临床基因疗法产品评估的监管发展.
科学领域:
- 毒理学和药理学 毒理学和药理学
- 基因治疗 基因治疗
- 监管科学 监管科学
背景情况:
- 目前评估基因疗法 (GT) 致癌性的方法需要改进.
- 行业和监管机构寻求其他评估策略.
- 一次共识会议讨论了GTs的基因毒性评估.
研究的目的:
- 在基因治疗的背景下定义载体遗传毒性.
- 为了确定GT基因毒性评估的不确定性和合适的毒理终点.
- 为制定非临床GT产品评估的监管准则提供信息.
主要方法:
- 召开了NC3Rs/UKEMS (伦敦,2023年3月) 组织的受邀专家会议.
- 促进了讨论,并就GT基因毒性的关键方面达成共识.
- 为未来的研究和监管指导提供文档建议.
主要成果:
- 在载体基因毒性的定义上达成共识.
- 在当前的基因毒性评估中确定了不确定性的关键来源.
- 为评估GTs定义了合适的毒理终点.
结论:
- 建议为改善基因疗法的基因毒性评估提供了一个框架.
- 达成的共识将为制定监管准则提供信息.
- 需要进一步的研究来完善GT产品的非临床毒理学评估.
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