Science and Research
Ocular genetic therapeutics research
We are developing safer, more precise gene therapies using advanced delivery technologies – aiming to prevent, and potentially reverse, vision loss caused by conditions such as inherited retinal diseases, age‑related macular degeneration and glaucoma.
Overview
Our Ocular Genetic Therapeutics team focuses on improving how gene therapies are delivered to the eye, with the aim of making treatments more effective, safer, and accessible to more people.
Gene therapies for eye disease commonly use adeno‑associated viruses (AAVs) to deliver therapeutic genes into retinal cells. These vectors have enabled major advances in the treatment of inherited eye diseases, as they are generally well tolerated, can support long‑lasting gene expression in the retina, and can be produced at clinical scale.
However, naturally occurring AAVs were not designed to treat human disease. Many struggle to efficiently reach the cells most affected in blinding conditions, particularly when using less invasive delivery approaches. As a result, current gene therapies may require higher doses, have limited precision or be unsuitable for some people.
Our research focuses on developing next‑generation gene‑delivery systems that overcome these challenges. By improving how therapeutic genes are delivered to specific retinal cells, we aim to unlock the full potential of gene therapy for a wider range of eye diseases, including inherited retinal diseases, age‑related macular degeneration and glaucoma.
Why this research is important
Many blinding eye diseases are caused by genetic changes that damage or destroy retinal cells, leading to progressive vision loss and, in some cases, blindness.
While gene therapy offers a powerful way to address the underlying cause of these conditions, current delivery technologies can limit who can be treated and how effective treatment can be.
Improved gene‑delivery systems have the potential to:
- Deliver therapeutic genes more efficiently and at lower doses
- Target the cells most affected by disease with greater precision
- Support less invasive treatment approaches
- Reduce the risk of immune‑related side effects
- Expand access to gene therapy to more people.
Our research is advancing safer, more precise gene‑delivery technologies to protect vision, slow or stop disease progression, and help restore sight for people with blinding eye conditions.
Key research questions
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- How can we design AAV vectors that efficiently reach hard‑to‑target retinal cell types, particularly using less invasive delivery methods?
- Can engineered vectors reduce the dose required while maintaining safety and long‑lasting gene expression?
- How can we improve gene delivery across retinal barriers to achieve more consistent treatment outcomes?
- How can we increase targeting precision to minimise off‑target effects?
- Can we develop gene‑delivery vectors that are less affected by pre‑existing immune responses, expanding eligibility to more people?