Stable macromolecule formulation

纳米颗粒给药系统（DDS）正因其极大地提高许多治疗方法的安全性和有效性的潜力而受到越来越多的关注。这些系统解决的主要挑战包括不溶性、稳定性差或具有显著脱靶效应的活性药物成分（api）。这对癌症治疗和新基因治疗尤其重要，因为癌症治疗通常具有高毒性，而新基因治疗不稳定，不能跨越生物屏障。通过在纳米载体中封装或以其他方式保持原料药分子，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分散的解决方案。特别是对于大分子，这些技术需要低温：也就是说，它们的储存在-80或-20摄氏度，以避免降解。这限制了这些分子在偏远地区的使用，但也增加了它们的碳足迹，并向净零、分散的医疗保健过渡。该项目将开发一种基于金属有机框架（mof）的新型DDS，这是一种具有非常广泛功能的材料，可用于车辆设计的模块化方法。它们几乎可以接受任何API，并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，就会与新的大分子表现相似；表面改性——一旦它的影响被表征出来——预计在应用于新的载体时也会有类似的表现。因此，mof可以为载体设计提供一种“模块化工具箱”方法，并且可能是最接近于真正的即插即用系统的技术。剑桥向量生物科学公司正在努力加速这项技术向临床的过渡。这个为期6个月的项目将通过提高siRNA在室温下的稳定性来证明该平台在新型siRNA基因疗法中的功能。它将针对临床相关里程碑，涵盖该技术的功效和生物相容性。在此过程中，它将验证制药行业的技术并降低风险，使合作能够结构化，平台的开发能够在项目完成后继续进行。该项目的重点是胰腺癌的分散式医疗保健。然而，该平台具有广泛的适用性，并且在项目后将开发相邻的、自我驱动的医疗保健领域。

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.