Current gene and cell therapy clinical trials for sight loss

We are translating our research on inherited and acquired forms of sight loss into clinical trials. Find out about how you can support our work and help develop effective therapies.

We are currently conducting two ground-breaking clinical trials:

Phase I/II trial to assess safety and efficacy of viral RPE65 gene therapy for Leber congenital amaurosis

We began the world's first gene therapy trial in the eye in 2007.

The trial is to test the safety and efficacy of gene therapy using an adeno-associated virus (AAV) to deliver the RPE65 gene to retinal cells in patients who have Leber congenital amaurosis (LCA).

Initial results published in 2008 (Bainbridge et al. NEJM 2011) suggest that the technique is safe and can restore night-vision in some cases.


Further patients have been treated, and the latest arm of the study includes younger patients.

The encouraging results from this trial suggest that gene therapy can be an effective treatment for inherited retinal degeneration, and extensive evidence from laboratory research shows that other diseases that lead to blindness may also be treatable using gene therapy.

Each of these therapies requires the development of a new gene therapy vector and independent safety testing

Pipeline of gene therapy treatments for sight loss

The results of this trial indicate that gene therapy in the eye is safe and can be effective at treating sight loss.

We are developing a pipeline of gene therapy strategies for a range of other conditions that cause sight loss.

Phase I trial to determine the safety of transplanting stem cell-derived retinal cells

We are conducting Europe's first clinical trial looking at the safety of injecting retinal cells, derived from embryonic stem cells, into the eyes of patients with Stargardt macular dystrophy.

Stargardt Disease
White flecks as seen in Stargardt disease


The cells being injected are retinal pigment epithelium (RPE) cells that were grown from human embryonic stem cells in a dish, by the American company Advanced Cell Technology Inc.

This trial involves patients who are already severely visually impaired and is designed to show whether the injection of these cells is safe - future studies will be required to determine if cell transplantation can restore sight.


Page last modified on 30 nov 12 10:50


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