Novel Gene Therapy May Prevent Blindness in Glaucoma Patients

InteliSpark client, Advanced Vision Technologies, wins a phase I STTR (Small Business Technology Transfer) grant from the National Eye Institute at the National Institutes of Health. Dr. John Danias, a clinician scientist, will lead this project in developing a novel gene therapy to treat high intraocular pressure (IOP), or high fluid pressure in the eye, that is known to increase the risk for both development and progression of blindness in glaucoma patients.

Glaucoma, a disease that damages the eye’s optic nerve, is the second cause of blindness worldwide with around 3 million Americans living with the disease currently. The economic impact of the disease on the US government is around $1.5 billion annually including health care costs, social security benefits and lost income tax revenues.

Open angle glaucoma (OAG), the most common form of the disease, results in increased eye pressure and is a leading cause of blindness. While there is no cure yet for Glaucoma, early detection and treatment may prevent further vision loss. Traditional therapies, in the form of eye drops, require regular administration over long periods of time which often leads to issues with patient adherence and managing the disease effectively. Furthermore, traditional therapies often do not address the underlying cause of the disease.

To meet the need for more effective treatments, Advanced Vision Technologies will target the underlying pathology of the disease in the outflow tissues by knocking down SERPINE1, a gene with critical effects in regulating outflow facility. If phase I is successful in confirming the gene therapy target, phase II will develop and test the efficacy of therapeutics to the outflow tissues in animal studies, thus setting the foundation for human clinical trials in the future. This treatment will address the issue of patient adherence found in traditional therapies by taking the patient out of the delivery system and may ultimately prevent vision loss for millions of patients across the country.