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.