To develop a novel drug loading strategy for exosomes with broad clinical applications

我们的项目旨在提高外体的药物装载效率，外体是由我们的细胞分泌的细胞外小泡，用于将分子货物从一个细胞运送到另一个细胞。外切体已经成为一种令人兴奋的药物传递载体，一些临床试验已经证明了外切体制剂的安全性和有效性。作为一种靶向给药策略，它们可以包装基于小分子、多肽和核酸的药物-增强体内药物的稳定性和细胞摄取，并具有工程化靶向的潜力。我们的技术将通过增加可伸缩性和减少治疗开发的异质性来改进基于外体的广泛疾病的治疗。我们的目标是通过产生一种我们称之为‘加载因子’的蛋白质平台来实现这一点。该项目将包括概念验证实验，以证明负载因数对此目的的有效性。

Our project aims to improve drug loading efficiency into exosomes - small extracellular vesicles which are secreted by our cells and used to traffic molecular cargo from one cell to another. Exosomes have emerged as an exciting drug delivery vehicle and several clinical trials have demonstrated both the safety and efficacy of exosome formulations. As a targeted drug delivery strategy they can package small molecule, peptide and nucleic acid based drugs - enhancing drug stability in the body and cell uptake as well as having the potential for engineered targeting.Our technology will improve exosome-based treatments for a broad spectrum of diseases through increasing the scalability and reducing the heterogeneity in development of the therapeutic. We aim to do this through generation of a platform of proteins we call 'loading factors'. The project will involve proof of concept experiments that demonstrate the efficacy of the loading factors for this purpose.