Gene therapy manufacturers cannot currently produce a crucial delivery vehicle—the high-capacity adenovirus—without contaminating it with the parent virus that is needed to make it. This contamination problem has blocked the use of these viral vectors for 40 years, even though they can carry much longer DNA sequences than standard gene therapy tools. Many genetic diseases require delivering a large piece of DNA or multiple pieces at once, and existing systems simply cannot fit them. The high-capacity adenovirus can, but only if it can be made safely for human use. The team has a patent-pending method to reduce contamination to acceptable levels. Over 18 months, they will demonstrate that the manufacturing process works at scale and validate the system for treating several diseases in the lab. If successful, the technology could open up gene therapies for conditions that currently have no treatment options. The business model involves licensing the method to gene therapy companies while running an in-house programme to prove the concept for a specific large-gene disorder. The ultimate output, if all goes to plan, would be new treatments for patients who currently have none.
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**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.
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