How early funding helped advance gene therapy for inherited eye conditions
1 April 2025
Early-stage funding plays a vital role in moving research from the laboratory to clinical practice. Thanks to donor support, Moorfields Eye Charity has been instrumental in advancing research, accelerating progress that might otherwise have struggled to gain momentum.
One example is the gene therapy research led by Professor James Bainbridge, professor of retinal studies at UCL and consultant ophthalmologist at Moorfields Eye Hospital where initial funding provided the early evidence needed to attract further investment.
Professor James Bainbridge
This support is essential for translating scientific discovery into treatments that will benefit patients in the future. Professor Bainbridge, who has spent over two decades at the forefront of genetic eye disease research, combines microsurgery with molecular biology to develop treatments.
He explained that seed funding from the charity was key in shaping a programme that progressed from research to clinical trials:
It’s been critical in enabling us to get projects started and gather pilot data to support applications for larger funding. This research is expensive, and without that initial support, it would be hard to move forward.
Gene therapy is one area in ophthalmic research that has seen remarkable progress, particularly in treating children born without functioning copies of genes essential for vision. Professor Bainbridge added:
It’s possible to improve outcomes by providing the retina with the gene that’s otherwise lacking. That can improve sight in the short term and may also help protect it in the longer term.
These advancements not only offer hope for rare inherited conditions but are also laying the groundwork for treatments that could benefit a much wider group of patients. Early, proof-of concept studies enabled by the charity have helped researchers gather the evidence needed to apply for major grants and attract commercial interest. As Professor Bainbridge explained:
That early support has helped us demonstrate potential and secure the larger-scale funding needed for clinical trials.
The benefits of this support extend beyond the research itself. “Students form a large part of our research teams,” he said. “They gain experience in translational research, and many go on to pursue careers in science, medicine, or industry.”
Some stay at Moorfields, while others apply their knowledge globally, improving treatments and diagnostics for eye health. Over the years, some treatments have been taken forward by biotechnology and pharmaceutical companies, moving closer to becoming widely available. Professor Bainbridge added:
It’s when it gets to that stage that you start to see the potential for real impact - not just for rare conditions, but also for more common diseases affecting many people.
Looking back on the progress made, Professor Bainbridge was highly positive:
I’ve been surprised how quickly the field has moved forward. I would not have imagined twenty years ago that the progress could be so fast.
He credits the long-term relationship with Moorfields Eye Charity as a key factor in the programme’s success.
There’s trust and an understanding of the value of early-stage research. Even a small contribution at the beginning of a project can have a big impact.
