IBioIC have funded a long-standing collaboration between AskBio Europe and Matthew Nolan’s group at the University of Edinburgh, where several breakthroughs have been made towards new gene therapy tools. Gene therapy is a potential treatment for many genetic diseases, where healthy DNA is inserted into patients’ own cells to treat diseases such as certain cancers or cystic fibrosis.

Targeting DNA to the right part of the body

It can be difficult to target the healthy DNA to the correct part of the body to treat a disease, so the project team worked on creating a library of small DNA elements, which they could test for their targeting abilities. The scientists were particularly interested in targeting DNA to the brain, which is one of the hardest areas to access for drugs and gene therapies.

New knowledge and methods

Creating the DNA library was challenging and required the project team to come up with innovative methods and solutions. The first challenge was to create an algorithm to identify all the small DNA parts that should be included in the library. The company generated significant expertise in developing this algorithm, further cementing their position in this field. The DNA parts then had to be synthesised and amplified. This required the development of a novel methodology, which the company was able to patent. The delivery strategy for these small DNA elements was also optimised, generating more knowledge for the company.

Successful targeting to the brain

Companies such as AskBio do not have their own animal facilities to test the effectiveness of a medication such as a gene therapy, so Edinburgh University was able to provide access to these facilities as part of the project. Using Matthew Nolan’s knowledge and Edinburgh’s facilities, the DNA library was injected into mice and the location of each library component was analysed. In this way, the team were able to identify which of the small DNA elements targeted the brain (and other tissues) and would therefore be useful to target treatments to the brain in future.

To hear more about project funding, contact projects@ibioic.com or visit our Funding page.