Public Funding for Refeyn Ltd

The therapeutic potential for gene therapies is transformative, where many largely intractable diseases will be treated safely, effectively and durably. Therapies are now starting to get approved (e.g. Roctavian, Zolgensma, and Luxturna), but cost remains a large barrier to entry and widespread patient access, partly due to suboptimal manufacturing processes. If gene therapies are ever going to be used to treat highly prevalent diseases, then breakthroughs need to be made in manufacturing. One area in need of improvement is process analytical technology (PAT) and continuous process monitoring. Current methods rely on laborious off-line analysis performed by highly trained operators at discrete time-points which introduces significant costs, delays and increases the chances of contamination. This project will use next-generation automation and novel PAT technologies to enable real-time characterisation and control of the manufacturing process.

Gene therapy holds huge promise for many conditions that have been difficult to prevent or treat using conventional drugs, including inherited, rare and age-related diseases, and cancers. As the industry grows, there is an urgent need for more efficient, effective technologies for quality control tests. Technologies developed for traditional drugs have major limitations when applied to complex gene therapies, such as adeno-associated virus (AAV) gene therapies, which use virus particles to deliver genes that can help treat or cure disease. For AAV therapies, one important factor is how many vector particles are carrying the target gene, but current approaches for measuring this are expensive and time-consuming, and usually need to be outsourced. This creates a processing bottleneck and means the measurement is usually done only at the end of production, even though such information is highly desirable throughout development and production. Separate assays are needed to measure contaminants, creating further inefficiencies. As another important limitation, no available technology can distinguish partially loaded particles from full ones. In this project, we propose to address these challenges using mass photometry, a technology we developed that measures the mass of single particles using minimal sample. We believe this technology could be a game-changer in this area because preliminary experiments show it can outperform the current state-of-the art. It can measure the percentage of loaded particles while simultaneously measuring amounts of smaller contaminants, and it is up to 15-fold faster and 100-fold cheaper than current approaches. Here, we plan to benchmark mass photometry against existing approaches and also make improvements, to better tailor our approach to the requirements of AAV vector characterisation. The project is a collaboration between Refeyn Ltd (the world's sole producer of mass photometry technology), who will develop the instrument, and the Cell and Gene Therapy Catapult, who will provide AAV material and conduct benchmarking. If successful, this project will deliver a transformative technology, helping accelerate the development, production, approval and safe use of promising gene therapies. It will advance the gene therapy sector, bringing economic and other benefits for the UK and beyond. Targeting a rapidly growing market with a superior, innovative technology, this project represents a sound investment. It is also timely; regulations are now being formed in this area, creating an opportunity to embed this technology in production and quality control processes.