Transforming genetic medicine for rare disease
We are the MRC Centre of Research Excellence in Therapeutic Genomics, bringing together scientists, clinicians, patients and partners to improve treatment for rare genetic disease.
We are developing flexible therapeutic platforms that can be adapted across many conditions, rather than one disease at a time. We begin with rare single-gene disorders affecting the blood, eye and brain, and use what we learn to help speed the path from diagnosis to treatment.
Our work spans five research themes and is grounded in collaboration, responsible research and a shared commitment to making genetic medicine ready for real-world use.
Welcome from the Director
When I established the MRC CoRE in Therapeutic Genomics in 2025, I did so with a bold and singular vision: to move genetic medicine from a series of high-profile "one-off" clinical anecdotes to an ordinary, customisable therapy platform. Our mission is to "make genetic medicines routine"—to build the open-source tools and de-risked delivery pipelines necessary to go from a patient's diagnosis to a bespoke therapy in just 100 days.
Professor Stephan Sanders
Director, MRC CoRE in Therapeutic Genomics
Latest news
Blood
Starting with primary immune deficiencies associated with Epstein-Barr virus (EBV), we will create a template for "how to deliver gene therapies": every step in the process from patient engagement and consultation, through to regulatory approval.
Brain
Aiming to move away from a "one-by-one" approach to choosing therapeutic targets for neurological diseases, and instead to develop a suite of flexible tools for gene regulation in the brain.
Eye
Aiming to develop and validate multiple, distinct gene therapy strategies for inherited retinal diseases (IRDs) and age-related macular degeneration (AMD), using in vivo gene editing techniques.
Genomics
The analytical engine of the MRC CoRE, providing a centralised, CoRE-wide toolkit and data framework.
Delivery
Working on a range of options to transport therapeutic agents across the blood-brain barrier (BBB), to treat neurological diseases.
