What is the MRC CoRE?

We know that many rare genetic disorders could be treated with current technology.  However, developing personalised genetic medicines is difficult and expensive.  At the current pace, it would take the scientific community 2,000 years to create all the therapies we believe are possible.

The MRC CoRE's job is to fix the whole system.  We want to change how genetic medicines are made and given to patients. Instead of creating one treatment for each disorder, we are building customisable "therapeutic platforms."  These are methods that can be adapted to treat many different disorders.

We will begin with a few example diseases that affect the blood, the eye and the brain.  The lessons we learn from these will help us make treatments for other genetic disorders.

We have seen that it is possible to create genetic medicines.  For example, a treatment for sickle cell disease was developed in 200, and bespoke medicines have been made for single patients.  As of May 2025 members of the MRC CoRE team showed that personalised gene-editing therapy can be created and delivered in just a few months. 

We also know that genetic therapies can be retargeted to treat a different disorder.  This opens the door to flexible, reusable gene therapy tools.

However: these successes are currently rare, and required huge effort from teams of hundreds of people.  Over the next 7-14 years, we aim to turn these lessons into a toolkit which can be more easily shared and used.

By drawing on the UK’s strengths in clinical genome sequencing, advanced manufacturing, and an independent regulatory system, The MRC CoRE will connect the dots and help place the UK at the forefront of advanced therapeutics.

Our approach is called ‘Therapeutic Genomics’. It is a genome-led, patient-centred, data-driven vision to bring genome-targeted therapies to the clinic at scale. 

Genome-targeted therapies such as CRISPR-Cas9, CRISPR-Cas13, base editing, ASOs and oligos have opened a new frontier in medicine.  For the first time, we can treat the underlying causes of disease, not just the symptoms.

The goal of the MRC CoRE TG is to take this even further: 

Rather than thinking of these therapies as specific treatments for individual conditions, we will turn them into flexible “therapeutic platforms”.  

The goal is a set of tools to change the genetic message, a set of vehicles to deliver the therapies, and knowledge to adapt and retarget them for new conditions.  

This toolkit will include data, computer models, expertise, delivery systems, assays and high-throughput processes for exploring and testing new therapies.

We are building a research culture that supports excellence in both science and people. 

As an MRC Centre of Research Excellence in Therapeutic Genomics, we champion a Team Science approach - focused on patients, driven by innovation and collaboration, and guided by open and responsible research practices. 

Together, we create an inclusive environment where diverse expertise delivers real impact for patients, researchers, and society.

To deliver our vision, we need to understand:

• Which variants and patients can be treated?
• How can therapeutic platforms be created and adapted for many disorders?
• How can these therapies be delivered safely and effectively?
• How can lessons from one therapy improve the next?
• What changes are needed in research culture and the clinical system to get therapies to patients?

The central funding for our project comes from the UK Medical Research Council (MR/Z504725/1 and MR/Z504877/1).  

We closely coordinate with projects supported by a raft of other funders including the Harrington Discovery Institute, Chan Zuckerberg Initiative, ARIA, BioMarin Pharmaceutical, the De Laszlo Foundation, the University of Oxford Public & Community Engagement with Research (PCER) Fund and others.