Viral Vector to Treat Age-Related Macular Degeneration and Other Eye Diseases

Technology #14807

Simple Injection Treats Photoreceptor Problems, Imparts Light Sensitivity to ON Bipolar Cells

This viral vector treats eye diseases through a safer, less invasive injection and could restore useful vision to individuals with a type of age-related macular degeneration that affects more than 2 million people. “Dry” age-related macular degeneration occurs when photoreceptors in the eye are damaged or begin to atrophy, resulting in vision loss. There are no available treatments capable of reversing “dry” AMD. University of Florida researchers have developed a viral vector that can be used to deliver a light-sensitive channel and impart light sensitivity to ON bipolar cells, the neurons immediately downstream from photoreceptors. This vector could also be used to restore light sensitivity to patients with inherited retinal disease that exhibit advanced photoreceptor degeneration. In addition, it could be used to treat diseases affecting bipolar cells, such as congenital stationary night blindness. Available vectors that target bipolar cells require a surgically invasive subretinal injection, which would further damage the retina in patients with AMD or advanced inherited retinal degeneration. This therapy can be administered with an intravitreal injection (injected through the vitreous part of the eye), which is safer, less invasive and could be performed in a standard outpatient setting.

Application

Therapy for eye diseases including age-related macular degeneration, inherited retinal degeneration and congenital stationary night blindness

Advantages

  • Administered through an injection into the vitreous of the eye, improving patient safety and comfort
  • Could be administered in clinic rather than a surgical suite, increasing accessibility to large patient populations
  • Restores useful vision to patients with age-related macular degeneration (AMD) or advanced inherited retinal disease who have no available treatment options

Technology

Researchers at the University of Florida have developed an AAV vector and cellular promoter combination that is capable of highly efficient and selective transduction of only ON bipolar cells. This tool can be used to deliver light-sensitive ion channels to these cells and restore the retina’s innate ability to register and process light signals even in the absence of photoreceptors. Imparting light sensitivity to ON bipolar cells which are closest to the natural start of the signaling cascade (photoreceptors) will result in the transmission of a more refined and “meaningful” signal to the brain, allowing for better visual acuity. The combination of an AAV capsid and specific cellular promoter will also allow the development of treatments for various forms of congenital stationary night blindness which are caused by defects in ON bipolar-specific genes.