Brain Research Theme

Our Brain research team is developing new approaches to treating neurological and neurodevelopmental conditions caused by genetic changes. Rather than developing a separate treatment for each condition, the team aims to create flexible technologies that can be adapted to target different genes and diseases.

The research focuses on regulating gene activity, either by reducing the production of harmful proteins or increasing the levels of proteins needed for healthy cell function. The team is investigating technologies including CRISPR-Lock, which allows precise control of RNA, and methods that increase protein production by modifying how genetic instructions are read. Researchers are also developing antisense oligonucleotides (ASOs), short molecules designed to alter how specific genes are expressed.

Artificial intelligence is supporting this work by helping researchers analyse existing ASO research and identify promising therapeutic approaches. The team is also developing CellXpress.ai, an AI-enabled platform designed to automate cell culture and testing, allowing potential treatments to be investigated more efficiently, consistently and at a larger scale.

Initial projects include developing ASOs that selectively target disease-causing gene variants associated with encephalopathy, a condition affecting brain function, and exploring ways to increase the activity of genes linked to neurodevelopmental delay.

The Brain team works closely with the Delivery research team to investigate how these experimental treatments can reach their intended targets in the brain, including overcoming the blood-brain barrier, which prevents many medicines from entering the brain. Together, this research aims to establish more adaptable approaches to developing genetic therapies for neurological conditions.