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The Gene Team

Funding from Retina UK has allowed to team to explore the viability of gene therapy as a treatment for RP and related conditions, and build a pipeline of clinical trials.

The Gene Team is led by Professors Robin Ali, James Bainbridge and Michel Michaelides at the UCL Institute of Ophthalmology and Moorfields Eye Hospital.

Find out more information about gene therapy.

So far, the team has:

  •  Completed the first clinical trial of RPE65 gene therapy to treat LCA2, one form of Leber congenital amaurosis, and secured funding from the Medical Research Council to conduct a further trial with a new optimised vector.
  •  Developed a gene therapy for LCA4, another type of Leber congenital amaurosis. The gene therapy vector will be tested in the coming months and should lead to a clinical trial at Moorfields Eye Hospital.
  •  Designed a clinical trial of CNGB3 gene therapy to treat one form of achromatopsia – the trial is expected to take place in the next two years.
  • Developed a gene therapy vector for X-linked RP3, one of the most common forms of RP, and designed a clinical trial. The trial is expected to start in the summer of 2017.

Their future plans include:

  • Continuing to develop new gene therapies for inherited retinal dystrophies, seeking funding from a range of sources to support pre-clinical research and early phase clinical trials.
  • Develop a commercial strategy to attract private investment in the future for the development of licensed gene therapy products.
  • Investigate a number of inherited retinal dystrophies that may respond to gene therapy, including Usher syndrome, Stargardt disease, LCA type 13, Batten disease and Bardet-Biedl syndrome.

Gene Team updates