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对遗传视网膜疾病的基因治疗临床试验的频率和模式

Hossein Ameri¹, Niranjana Kesavamoorthy^2,3, Dara N Bruce⁴

¹Department of Ophthalmology, USC Roski Eye Institute, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. ameri@med.usc.edu.

Advances in experimental medicine and biology

|February 10, 2025

概括

对遗传性视网膜疾病 (IRD) 的基因治疗临床试验显示出显著的进展,到2022年底将有101项试验. 腺相关的病毒载体占主导地位,视网膜色素炎是最有针对性的疾病.

科学领域:

眼科医生眼科眼科
遗传学是一个遗传学.
临床研究临床研究

背景情况:

在全球范围内,遗传性视网膜疾病 (IRD) 是导致视力丧失的重要原因.
基因疗法为这些遗传疾病提供了一个有前途的治疗途径.
对IRD基因疗法临床试验的全方位数据对于未来的研究和开发至关重要.

研究的目的:

系统地审查和分析全球基因疗法临床试验遗传性视网膜疾病 (IRD) 进行到2022年底.
确定治疗方法的趋势,试验阶段,地理分布和目标IRD亚型.
为IRD提供基因治疗领域当前的现状和进展的概述.

主要方法:

从15个国际登记处收集数据,其中包括clinicaltrials.gov.gov.
对针对IRD的101个基因疗法临床试验的分析.
根据治疗方法对试验进行分类 (基因增强,抑制性RNA等). ),临床阶段,地理范围和特定的IRD目标.

主要成果:

到2022年底,共有101个IRD基因疗法试验被确定为2022年底.
使用病毒载体,特别是腺相关病毒 (AAV) 的基因增强是主要的方法 (77个试验).
视网膜色素炎 (39个试验) 和RPE65介导的视网膜缩 (13个试验) 是最常见的向疾病. 美国在审判进行方面处于领先地位.

结论:

针对IRD的基因疗法已经出现了大量的全球临床试验活动,基因增强和AAV载体是主要的策略.
该领域正在进步,FDA批准的一种治疗方法和大多数早期到中期阶段 (阶段1/2) 的试验证明了这一点.
持续的研究和跨国合作对于进一步开发更广泛的IRD有效基因疗法至关重要.

关键词:

临床试验中的临床试验.基因增强是一种基因增强.基因治疗是一种基因疗法.遗传性视网膜退化遗传性视网膜退化利伯的先天性黄斑症 (Leber congenital amaurosis) 是一种先天性黄斑症.勒贝尔遗传性视神经病变 (Leber hereditary optic neuropathy) 是一种遗传性的视神经病变.视觉遗传学视觉遗传学视网膜色素炎 (Retinitis Pigmentosa) 是一种可见的疾病.病毒载体病毒载体

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