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    Gene therapy new frontier for ocular disorders

    Ongoing trials have potential to transform, offer long-term therapy for diseases

     

    There are currently no treatments for LCA, Dr. Kiss noted, but several companies are developing gene therapies to deliver a functional RPE65 gene to restore vision. At least one pivotal phase III trial using gene therapy to treat LCA will likely be submitted to the FDA for approval later this year.

    Defending OD-performed surgery

    All of the key ingredients needed for successful ocular gene therapy are in place, Dr. Kiss noted. Researchers have developed a menu of successful vectors that target different layers of the retina to deliver genes to treat different diseases. They have identified target genes responsible for specific diseases, have created successful animal models, surgical approaches, outcomes measures and safety data.

    AAVs that target the nerve fiber layer may be useful for the treatment of glaucoma while vectors that target the Muller cells could treat X-linked retinoschesis. Vectors that transfect photoreceptors may be useful for some forms of RP while other vectors that transfect the RPE and choroid are more appropriate for other forms of retinal degeneration as well as wet and dry AMD.

    Current approaches deliver therapeutic vectors via intravitreal or subretinal injection. The vector is a protein envelope, or capsid, with its native DNA replaced by the target gene and a promoter. The promoter enhances the activity of the gene to boost production of the target protein. To treat a genetic disorder such as LCA, the target gene produces the missing protein responsible for disease, RPE65 in this case. To treat acquired disorders such as neovascular age-related macular degeneration, the target gene produces a therapeutic protein such as an anti-VEGF molecule sFLT.

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    Both the intravitreal and subretinal approaches can offer efficient transduction and long-term expression of the target protein. Vectors are non-pathogenic, have low immunogenicity, are non-replicating and do not integrate with the ocular genome. 

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