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Both inherited and acquired ocular diseases are potential targets for gene therapy. NAV™ Technology has been shown to be very efficient at gene delivery to the retina, so initial work is focused on retinal diseases including the inherited diseases Leber congenital amaurosis and retinitis pigmentosa, and the acquired disease wet age-related macular degeneration (wet AMD).
The challenges in treating ocular diseases are:
- Directing gene expression to the appropriate cell types, both by the route of injection in the eye, and by the dose and selection of vector.
- Getting high enough levels of expression of optimal therapeutic benefit. Because the volume that can be injected is limited, gene therapy vectors that are highly efficient are desirable, such as the NAV Technology, which has been extensively screened in the eye.
The eye is a very active area of clinical investigation in gene therapy. Several clinical trials have been performed in patients with Leber congenital amaurosis, an inherited form of blindness. In these patients, rAAV2 was injected subretinally, and partially restored vision in the treated eyes, demonstrating both safety and efficacy even in the phase 1 clinical trials.
Phase 1 studies are ongoing in wet AMD, in which the expressed transgenes are designed to inhibit the formation of new, leaky blood vessels. One trial uses a lentivirus delivered subretinally, and the other uses an rAAV2 injected intravitreally.
For some of the ocular disorders, especially those in which the therapeutic transgene encodes a protein that is not normally expressed in the eye, tight control of the levels of therapeutic transgene expression will be needed for optimal efficacy and safety. In addition, in patients in whom the therapy may last decades, the ability to withdraw the therapy if needed provides an additional essential safety feature. In these applications, our NAV platform includes a gene regulation technology that will allow for pharmacologic control of the timing and level of gene expression.
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