对商业性寡核酸药物产品的审查
Bhavani Prasad Vinjamuri1, Jiayi Pan1, Paul Peng1
1Pharmaceutical Operations & Technology, Biogen, 225 Binney Street, Cambridge, MA 02142, United States.
Journal of pharmaceutical sciences
|April 28, 2024
概括
本综述分析了20种已批准的寡核酸药物,包括反感性寡核酸 (ASO) 和小干扰性核酸 (siRNA). 这些发现提供了一个通用的配方起点,以加速早期药物开发.
科学领域:
- 制药科学 制药科学
- 药物开发 药物开发
- 生物技术是生物技术.
背景情况:
- 寡核酸治疗药物代表着快速增长的药物类别.
- 了解配方和输送属性对于成功的药物开发至关重要.
- 商业化需要强大的配方策略,用于各种管理路径.
研究的目的:
- 在美国和欧盟审查和分析商业上已批准的寡核酸药物产品.
- 为了确定常见的配方特征,施用途径和寡核酸药物的包装.
- 为早期的寡核酸药物开发提出一个通用的配方起点.
主要方法:
- 对20种已批准的寡核类药物产品进行系统审查.
- 对药物成分,度,pH值,施用途径和初级包装的分析.
- 数据汇编以确定共同的属性和趋势.
主要成果:
- 确定了20种产品:1种阿巴,12种反感性寡核酸 (ASO),6种小干扰性核酸 (siRNA) 和1种混合物.
- 典型的配方含有具有缓冲,pH调节和调节强度剂的寡核酸.
- 产品是溶液 (2.1-200 mg/mL,pH 6-8.7),主要用于静脉注射 (IV) 和皮下注射 (SC) 途径,使用瓶子和预填注射器 (PFS) 作为主要包装.
结论:
- 可以为IV,SC和内 (IT) 途径的寡核酸药物的早期开发提出一个通用配方起点.
- 对药物产品属性的协调评估有助于平台的概括.
- 这种方法可以加速早期的开发阶段,包括首次在人类身上进行的研究.
关键词:
在ASO的基础上,ASO是ASO.亚普特美尔 (Aptamer) 是一种药物.商业商业的商业.药物产品是一种药物产品.配方 配方 是一种配方.有氧核酸 (Oligonucleotide) 是一种包装 包装是一种包装.管理的途径.稳定的稳定性 稳定的稳定性siRNA 是一个RNA.更多相关视频
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