Stable macromolecule formulation

纳米药物递送系统(DDS)因其极大地提高许多治疗药物的安全性和有效性而受到越来越多的关注。这些系统解决的关键挑战包括活性药物成分(API)不溶、稳定性差或具有显著的脱靶效应。这些对于癌症疗法和新的基因疗法尤其重要，前者往往毒性很高，后者不稳定，无法跨越生物障碍。通过将原料药分子包裹或以其他方式保持在纳米载体中，治疗药物可以获得广泛的有益特性，包括缓慢和靶向释放。然而，目前的技术提供了非互补性和高度碎片化的解决方案。特别是对于大分子，这些技术需要低温：即它们在-80或-20摄氏度下储存，以避免降解。这限制了此类分子在偏远地区的使用，但也增加了它们的碳足迹，并过渡到净零、分散的医疗保健。该项目将开发一种基于金属有机骨架(MOF)的新型DDS，这类材料具有非常广泛的功能和模块化的车辆设计方法。它们几乎可以接受任何API，并可以根据需要使用行业标准技术进行修改。这意味着，MOF载体一旦被证明具有特定的大分子，将与新的载体表现出类似的行为；而表面修饰--一旦其影响被表征--预计将应用于新的载体时表现出类似的行为。因此，MOFS可能会提供一种“模块化工具箱”的载体设计方法，而且可能是迄今为止最接近真正的即插即用系统的技术。载体生物科学剑桥大学正在努力加快这项技术向临床的过渡。这个为期6个月的项目将寻求通过提高新的siRNA基因疗法在室温下的稳定性来展示该平台在各种新型siRNA基因疗法中的功能。它将以临床相关的里程碑为目标，涵盖该技术的有效性和生物兼容性。在这样做的过程中，它将验证这项技术并将其降低到制药业的风险中，从而使协作得以组织起来，并在项目完成后继续平台的开发。该项目的重点是分散治疗胰腺癌的医疗保健。然而，该平台具有广泛的适用性，相邻的、自主驱动的医疗领域将在项目后开发。

Nanoparticle drug delivery systems (DDS) are seeing growing attention for their potential to drastically improve the safety and efficacy of many therapeutics. Key challenges these systems address include active pharmaceutical ingredients (APIs) that are insoluble, have poor stability or have significant off-target effects. These are particularly important for cancer therapies, which are often highly toxic, and novel gene therapies, which are unstable and cannot cross biological barriers. By encapsulating or otherwise holding API molecules within a nanocarrier, therapeutics can be given a wide range of beneficial properties, including slow and targeted release.Current technologies, however, provide non-complementary and highly fragmented solutions. For macromolecules, in particular, these technologies require cryogenic temperatures: this is, their storage at -80 or -20C to avoid degradation. This limits the usage of such molecules in remote locations but also increases their carbon footprint and the transition to net-zero, decentralised healthcare.This project will develop a novel DDS based on metal-organic frameworks (MOFs), a class of materials with exceptionally broad functionality and a modular approach to vehicle design. They can accept virtually any API and be modified as required using industry-standard techniques. This means that a MOF carrier, once proven with a particular macromolecule, will behave similarly with a new one; and a surface modification -- once its impact has been characterised -- is expected to behave similarly when applied to a new carrier. MOFs may therefore offer a "modular toolbox" approach to carrier design and potentially the closest technology yet to a true plug-and-play system.Vector Bioscience Cambridge is working to accelerate the transition of this technology to the clinic. This 6-month project will seek to demonstrate the functionality of the platform across novel siRNA gene therapies by improving their stability at room temperature. It will target clinically relevant milestones covering the technology's efficacy and biocompatibility. In doing so, it will validate and de-risk the technology to the pharmaceutical industry, enabling collaborations to be structured and the development of the platform to continue once the project is complete. The focus of this project is de-centralised healthcare in pancreatic cancer. However, the platform has broad applicability and adjacent, self-driven healthcare areas will be developed post-project.