Delivery Research Theme

Our Delivery research team is developing new ways to transport genetic therapies into the brain, addressing one of the major challenges in treating neurological conditions. The brain is protected by the blood-brain barrier (BBB), a natural defence system that prevents many substances, including potentially beneficial medicines, from entering brain tissue.

The team is investigating specialised delivery vehicles, known as vectors, that can carry therapeutic genetic material across this barrier and into specific regions of the brain. An initial focus is on recombinant adeno-associated viruses (rAAVs), which are modified viruses designed to deliver genetic material into cells.

Researchers will screen and test a library of different rAAV capsids, the outer protein shells of these viruses, to identify those most effective at delivering therapies to targeted brain regions, including the cortex and striatum. These areas are involved in functions such as movement, learning and cognition.

Working closely with the Brain research team, the Delivery team aims to develop more effective methods for transporting experimental gene therapies to the cells where they are needed. This research could support the development of new treatments for a range of neurological and neurodevelopmental conditions.