Research themes

Therapy applications for blood, eye, and brain (using a variety of gene therapy techniques) 

Delivery vehicles, to get the gene therapies into the cells where they are needed 

Genomics tools for handling data, finding patients, and predicting 

• whether an individual’s condition is treatable with our current tools
• which combination of tools might be most effective
• which potential unwanted effects we should take extra care to avoid

We also have five cross-cutting themes, which inform all the work we do

Patient and Public Involvement and Engagement - we need to work in partnership with patients, their families, and the wider community.

Training - we need to support researchers to unlock their potential, use all their talents and learn to work as Team Scientists.

Regulatory Engagement - the Therapeutic Genomics approach is a completely different way of thinking about medicines.  We need to work with regulators to find ways to deliver these new therapies where they are needed, safely and quickly.

Health Economics - we need to ensure our therapies can actually be delivered by local health services and covered by health insurance.

Equality, Diversity and Inclusion - we need therapies for all, by all.