A new Cambridge spin-out is testing whether porous crystals called metal-organic frameworks can ferry cancer-killing RNA molecules directly into tumours, bypassing healthy tissue. Current nanoparticle drug carriers struggle with a fundamental problem: most of the dose ends up in the wrong place. They also tend to be one-trick ponies, each designed for a specific payload and a specific challenge. Metal-organic frameworks (MOFs) are different. These sponge-like crystals can be loaded with almost any therapeutic cargo—insoluble drugs, fragile proteins, or RNA—and their surface can be modified using standard industrial methods to control where and when the cargo is released. The company, Vector Bioscience Cambridge, is focusing first on pancreatic cancer, a notoriously hard-to-treat tumour, using siRNA molecules that can silence cancer-driving genes. If the platform works, it could transform drug delivery from a fragmented collection of bespoke solutions into a single, versatile system. That would mean faster development timelines for new RNA-based therapies, fewer side effects for patients, and a practical route to treat cancers that currently have no effective options.
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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.
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