A revolutionary, highly versatile drug delivery platform based on Metal-Organic Frameworks

纳米颗粒给药系统（DDS）因其极大地提高许多癌症治疗药物的疗效和安全性的潜力而受到越来越多的关注。DDS解决的关键挑战包括不溶性、稳定性差或具有显著脱靶效应的货物。使用目前的DDS技术，只有一小部分给药剂量最终到达靶部位以产生预期效果。此外，这些提供了非互补和高度分散的解决方案，个别方法只能解决这些挑战中的一部分潜在货物。金属有机框架（MOFs）于20年前首次开发，是近年来材料科学中最令人兴奋的领域之一。这些多孔混合固体在很大程度上是有效载荷不可知的，并且用于控制载体行为的修饰在不同的MOF之间很好地转移。这使它们能够容纳几乎任何治疗有效载荷，并使用行业标准技术来修改递送特征，提供一个技术平台，最终有可能成为药物递送行业急需的几乎通用的解决方案。Vector Bioscience剑桥成立于2021年，基于剑桥大学15年的研究，目标是成为第一家将这种极具前景的DSS平台推向市场的公司，专注于大分子递送。siRNA等大分子可能是现有最强大的抗癌药物，但目前还没有有效的方法将它们特异性地递送到肿瘤。GENERA通过验证一种具有巨大潜力的新的递送方法来响应Pathfinder Challenge对新型RNA递送方法和疗法的呼吁，以提高基于RNA的疗法的安全性和有效性。我们的第一个用例包括部署siRNA用于治疗难以治疗的癌症类型，从胰腺癌开始。

Nanoparticle drug delivery systems (DDS) are seeing growing attention for their potential to drastically improve the efficacy and safety of many cancer therapeutics. Key challenges DDSs address include cargos that are insoluble, have poor stability or have significant off-target effects. With current DDS technologies, only a small fraction of the administered dose ends up reaching the target site to have its intended effect. In addition, these provide non-complementary and highly fragmented solutions, with individual approaches that are only able to address a subset of these challenges with a subset of potential cargos. Developed for the first time 20 years ago, metal-organic frameworks (MOFs) are one of the most exciting areas in recent materials science. These porous hybrid solids are largely payload-agnostic, and the modifications used to control the carrier’s behaviour transfer well between different MOFs. This enables them to house virtually any therapeutic payload and use industry-standard technologies to modify the delivery profile, offering a technology platform with the potential to ultimately become the almost universal solution highly needed in the drug delivery industry. Vector Bioscience Cambridge was founded in 2021 based on +15 years of research at the University of Cambridge with the objective to become the first company to take this highly promising DSS platform to the market, focusing on macromolecule delivery. Macromolecules such as siRNA are potentially the most powerful anti-cancer drugs that exist, but there is currently no efficient way to deliver them specifically to the tumour. GENERA answers the Pathfinder Challenge call for novel RNA delivery methods and therapies through the validation of a new delivery method with enormous potential to improve the safety and effectiveness of RNA-based therapies. Our first use case consists of the deployment of siRNA for the treatment of hard-to-treat cancer types, starting with pancreatic cancer.