In 2024, the Princess Royal opened the Gene Therapy Innovation and Manufacturing Centre (GTIMC) at the University of Sheffield, a pioneering facility with a main mission to save lives. I’m a gene therapy researcher and the centre’s Director.
But I almost walked away from it.
This is how I turned the biggest disappointment of my career into a new era for UK gene therapy manufacturing…
14 years ago my team at the University of Sheffield developed a gene replacement therapy for the childhood form of Motor Neuron Disease, Spinal Muscular Atrophy (SMA).
We were the first to develop a proof-of-concept for life-saving treatment for the degenerative genetic disease. For the first time families receiving a heart-breaking diagnosis had hope.
No capacity:
I’d secured funding from the Medical Research Council (MRC) to manufacture the therapy. But everywhere I went in the UK it was the same answer - no capacity, no capacity, no capacity.
By this time, our US competitor - with a similar product, for the same disease - manufactured their therapy. Their product is now available to children and babies across the world.
After dedicating more than a decade to this research, our hard work would remain confined to the lab because of a lack of manufacturing capacity.
The UK is an international leader in biomedical research, but how can we transform patient care if we can’t manufacture the treatments that come out of this?
I had three options:
1. Stop doing translational research
2. Leave the UK to work somewhere that has capacity to manufacture therapies
3. Stay in the UK and do something about it
I chose the third and probably the most challenging option.
Home Truths:
I and other UK colleagues began lobbying and discussed with the funding bodies to share our frustration. We put it bluntly to them - ‘You’re spending all of this money on discovery programmes, but little/nothing is coming out of the UK.’ To their credit they listened.
They asked me to share my experience and disappointment.
And used it to secure funds to establish a new £18 million network of Innovation Hubs for Gene Therapies, dedicated to advance the clinical development of new genetic treatments through Good Manufacturing Practice (GMP), alongside translational support and regulatory advice.
The network - created by the MRC and LifeArc, with the Biotechnology and Biological Sciences Research Council (BBSRC) - opened facilities at the University of Sheffield, King’s College London and the NHS Blood and Transplant in Bristol.
The University of Sheffield, already renowned for its world-class translational research, has emerged as one of the leading players in gene therapy.
We’re in a new era for life-changing gene therapies in the UK and I’m so proud Sheffield is at the forefront.
To find out more about the Royal opening, you can read more here.
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