Coming Soon

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227,723
2025-04-01 to 2026-09-30
Collaborative R&D
**The Challenge:** Gene therapy and gene editing (e.g. CRISPR) are transforming the lives of patients with a range of previously intractable diseases, such as hemophilia. However, many challenges remain, and for some conditions there is no viable tool in the arsenal of the the genetic engineer. Often treating a disease requires the delivery of a long piece of DNA that simply will not package, or 'fit', inside the industry standard systems. Similarly, if you need to deliver multiple pieces of DNA, the same limitations apply. Within the industry though, tools are available that can resolve these significant needs. Unfortunately though, they cannot be manufactured safely and at large scale without becoming contaminated. **Our Solution:** Our project aims to fix these industry wide limitations by developing a new method to manufacturing a specific viral vector called a high-capacity adenovirus. These viral systems have existed for over 40 years, but have never come close to being used to treat patients because manufacturing them always results in contamination by a parent virus, adenovirus, that is needed to make them. We have patent pending on a new approach that will allow us to reduce the contamination down to acceptable levels, making our new method suitable for human medical usage. This should open up many new treatments for a range of currently untreatable human diseases. **Our Project:** Using Innovate funding, our project will run over 18 months and aims to fully demonstrate and exemplify this novel technology and validate its benefits. We will then show that the system can be used for a range of disease treatments in the laboratory and seek to publish the work and market its benefits to potential licensees and collaborators. **The Outputs:** Our business model will be a combination of high value non-exclusive licences to gene therapy and gene editing companies for diverse human and veterinary disorders, whilst simultaneously running an in house programme to demonstrate proof of principle for a large gene disorder. Data generated can be added into our patent filing during the next 12 months. We will also publish in a high profile publication (similar to our previous publication in Nature Communications) and present updates at international conferences/trade shows. Ultimately, over the longer term, the main output will be new and improved treatments for patients with currently little to no treatment options.