Lipid nanoparticle formulations for targeted local delivery

用于靶向局部递送的脂质纳米颗粒制剂

• 批准号: 2738552
• 金额: --
• 依托单位: University College London
• 依托单位国家: 英国
• 项目类别: Studentship
• 财政年份: 2022
• 资助国家: 英国
• 起止时间: 2022 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=studentship-2738552
• 关键词: Lipid; nanoparticle; formulations; targeted; local

Lipid nanoparticles (LNPs) can provide a safe, effective and stable delivery system for therapeutic agents such as mRNA. Intramuscular administration of LNP-mRNA vaccines has been used widely in the treatment of infections such as SARS-CoV-2 and intravenous LNP delivery of therapeutic mRNA has also been tested clinically to treat cancer and genetic disorders. This project aims to explore ways to modulate LNP-mRNA formulation for local administration to specific tissues, extending the utility of this powerful therapeutic approach to the treatment of acute local tissue and organ damage in areas of unmet clinical need.Main objectives within the project are as follows:1. Modulate LNP composition to optimise stable encapsulation of specific mRNA molecules2. Characterise the relationship between LNP lipid structure and organ/cell-specific delivery3. Use peptides to target LNPs to specific cells of interest4. Test selected optimal LNP formulations using relevant in vitro and in vivo modelsThe project has been developed to address the first of the EPSRC's Healthcare Technologies grand challenges; developing new therapies: focusing on the need to produce safer, more targeted treatments fit for the demands of the decades up to 2050 and beyond and is aligned primarily with the Advanced Product Design theme within the CDT.

脂质纳米粒(LNPs)可以为信使核糖核酸等治疗剂提供安全、有效、稳定的给药系统。肌肉注射LNP-mRNA疫苗已被广泛用于治疗SARS-CoV-2等感染，静脉注射LNP也已被用于治疗癌症和遗传疾病的临床试验。这个项目旨在探索如何调整局部给药的LNP-mRNA配方，以扩大这一强大的治疗方法的用途，以治疗未满足临床需要的地区的急性局部组织和器官损伤。该项目的主要目标如下：1.调整LNP的组成，以优化特定mRNA分子的稳定包裹2。描述LNP脂结构和器官/细胞特异性递送之间的关系3。使用多肽将LNPs定位于感兴趣的特定细胞4。使用相关的体外和体内模型测试选定的最佳LNP配方该项目的开发旨在应对EPSRC的医疗保健技术的第一个重大挑战；开发新的疗法：重点关注生产更安全、更有针对性的疗法的需求，以满足直到2050年及以后的几十年的需求，并主要与CDT内的高级产品设计主题保持一致。