Background

Equine recurrent uveitis (ERU), also known as moon blindness, is an autoimmune ocular disease and the most common cause of blindness in horses worldwide. It is estimated that approximately 25% of equine population is affected by this sight-threatening disease. Uveitis is a recurrent, chronic inflammation of the uveal tract in the eyes that usually starts with disruption of the blood-ocular barrier, allowing inflammatory immune cells to infiltrate, and then progresses to more destructive changes such as ocular cloudiness, cataract formation and eventually blindness.

ERU is currently treated through daily use of topical corticosteroids. The drawbacks of cortisone therapy are that it is not specific, requires several treatments, and can have adverse effects such as the development of corneal degeneration, glaucoma, cataract, ocular hypertension, bacterial infections, and laminitis. Consequently, a long-term and specific ERU treatment for horses is an unmet need.

Technology Overview

Researchers at North Carolina State University and The University of North Carolina at Chapel Hill have developed an AAV (Adeno-Associated Viral) gene therapy for the treatment of equine recurrent uveitis. This therapy uses a single intravitreal injection of adeno-associated virus to deliver a cDNA encoding an equine cytokine gene. Researchers found that horse T lymphocytes activity is suppressed in vitro when treated with this cytokine target. To test this approach in vivo, researchers injected experimental autoimmune uveitis (EAU) rat models with this novel AAV treatment and found that the occurrence of uveitis was significantly reduced. This improvement is associated with lower inflammatory cells infiltration into the vitreous body and retina after receiving AAV gene therapy.

Together these studies suggest that this AAV gene therapy targeting the equine cytokine can reduce ERU by alleviating the ocular inflammation, T cell infiltration and T cell activities, and this treatment can potentially mediate ERU long-term after just one injection. AAV gene therapy has been shown to be safe and well tolerated in general, and the researchers conducted chronic safety studies of this particular gene therapy product in normal rat eyes. No significant adverse effects were observed. Overall, this novel AAV gene therapy overcomes the challenges of current ERU treatments as this approach is specific, may offer long-term relief, and has no significant adverse side effects.

For more information, please click here to view the news article titled “Autoimmune uveitis targeted by AAV gene therapy”

Benefits

One treatment may have a life-long effect
Specific and effective
Cost-effective
Safe

Applications

The method can potentially be used to treat equine recurrent uveitis (ERU), also known as moon blindness, in horses.

Opportunity

NC State University is seeking an industry partner to license and commercialize the technology.

AAV Gene Therapy for Moon Blindness in Horses

IP Status

Seeking