对于稳定脂质纳米颗粒的大批量制造工艺:一个高度可定制的RNA输送平台
Suzanne Saffie-Siebert1, Nissim Torabi-Pour1, Andrew Gibson1
1SiSaf Ltd, Surrey Research Park, Guildford GU2 7RE, UK.
Molecular therapy. Methods & clinical development
|September 6, 2024
概括
稳定混合脂质纳米粒子 (sshLNPs) 提供了改善的RNA稳定性和保质期. 一个新的可扩展的制造工艺现在使这些先进的纳米载体用于RNA疗法的大规模生产成为可能.
科学领域:
- 生物技术是生物技术.
- 纳米医学是一种纳米医学.
- 药物运输 药物运输 药物运输
背景情况:
- 脂质纳米粒子 (LNP) 对RNA疗法至关重要,但在RNA稳定性和保质期方面面临挑战.
- 稳定混合脂质纳米粒子 (sshLNPs) 提供增强的稳定性和后期RNA加载能力.
- 以前的sshLNP生产方法不适合大规模制造.
研究的目的:
- 为稳定混合脂质纳米粒子 (sshLNPs) 开发一个可扩展的制造工艺.
- 为了使sshLNP的多公斤批量生产为临床翻译.
- 与小规模方法相比,确保大规模生产的sshLNP的物理和功能等价性.
主要方法:
- 开发了一种使用溶剂注射混合用于纳米粒子形成的修订协议.
- 集成的流入挤出用于多升体积.
- 优化了接流过,以去除剩余的有机溶剂.
主要成果:
- 成功建立了一个制造工艺,使得2公斤的sshLNPs.成功完成批量生产.
- 证明大规模生产的sshLNP具有与小规模批量相当的物理和功能性质.
- 新的工作流支持良好制造实践 (GMP) 协议.
结论:
- 一个可扩展的,基于溶剂注射的sshLNP制造工艺已经成功开发出来.
- 这一进步促进了sshLNP技术在RNA疗法中的临床转化和大规模部署.
- 优化的流程确保了sshLNP的质量和有效性,用于未来的治疗应用.
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