Gene Therapy

We are developing gene therapies for a wide range of ocular conditions.


1. Our centre has identified more inherited retinal dystrophies than any other centre worldwide and we are now building a comprehensive gene therapy programme to treat many of these disorders.

2. To develop gene therapy for age-related macular degeneration (AMD) and corneal disorders.

AIM 1 - We have developed a pipeline of therapies to treat inherited retinal dystrophies and have established three on-going clinical trials: LCA 2 due to defects in RPE65; achromatopsia due to defects in CNGB3 and X-linked RP due to defects in RPGR. We are now preparing additional trials for other inherited retinal dystrophies.

AIM 2 - We have established proof-of-concept for the treatment AMD and corneal disorders using gene therapy and are now developing pathways to clinical trials.