MRC Centre of Research Excellence in Therapeutic Genomics

Eight out of ten rare diseases have a genetic cause, and most genetic conditions are classified as rare. These diseases are often chronic and life‑threatening, with diagnosis frequently being a lengthy process. Most currently have no available treatment, and developing personalised genetic medicines is often difficult and expensive.

Genome‑targeted therapies such as CRISPR‑Cas9, CRISPR‑Cas13, base editing, antisense oligonucleotides (ASOs) and other oligonucleotide‑based therapies have opened a new frontier in medicine. For the first time, we can treat the underlying causes of disease, not just the symptoms. The Medical Research Council Centre of Research Excellence (MRC CoRE) in Therapeutic Genomics project aims to change how genetic medicines are developed and delivered to patients. Instead of trying to create a standalone treatment for each disorder, we are building customisable “therapeutic platforms”.

The MRC CoRE is led by the University of Oxford, bringing together a multinational and multidisciplinary team to deliver this project. The work spans disease areas affecting the brain, blood and eye, initially focusing on inherited retinal diseases, age‑related macular degeneration, primary immune deficiencies and neurological conditions such as Huntington’s disease.

The health economics component of the MRC CoRE assesses the costs and cost‑effectiveness of therapies such as CRISPR and ASOs across different diseases. It also explores alternative reimbursement mechanisms for healthcare payers and examines the preferences of key stakeholders, including patients and parents, using choice‑modelling methods.

Funder: Medical Research Council, part of UK Research and Innovation