Gene Therapy for Eye Diseases Including Uveitis and Diabetic Retinopathy

Technology #14556

Safer, One-Time Injection Reduces Inflammation Associated With Common Eye Diseases

This viral vector treats inflammation-associated eye diseases through a safer single injection. Inflammation causes pain and reduced vision in many eye diseases such as diabetic retinopathy, which affects more than 10 million people. Inflammation can serve a useful purpose, helping to remove potentially harmful materials from an injury site. Uveitis, age-related macular degeneration, and diabetic retinopathy are all associated with the excessive production of inflammasome activated cytokines, the proteins that signal for inflammation. Researchers at the University of Florida have developed a viral vector that controls the production of these inflammasome activated cytokines. Available treatments for this inflammation and inflammatory damage are invasive and increase risk of infection or other complications such as glaucoma. This viral vector could be provided in a single injection, restoring vision to patients without the risks of surgery or immune suppression.


Therapy for eye diseases including uveitis, age-related macular degeneration, and diabetic retinopathy


  • Administered through single injection, improving patient safety and comfort
  • Does not induce immune suppression, avoiding infection risks of available treatments


When the body is injured, it recruits inflammatory cells to the site of the injury. This inflammation, signaled by the protein Interleukin-1beta (IL-1β), is generally an attempt by the body to remove harmful cells or materials from the injury. In patients with uveitis and several other inflammasome-mediated diseases, IL1β production is unchecked. Unregulated inflammation can cause severe problems, especially in the eye. This adeno-associated viral (AAV) vector inhibits the production of active IL-1β by inhibiting caspase-1, which is needed to activate the precursor form of IL-1β.