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Retinal Disease

Aerie’s retinal therapeutics development programs are focused on significant retinal diseases, including age-related macular degeneration (AMD) and diabetic macular edema (DME), that represent major causes of sight-impairment and blindness in adults worldwide.

AMD is a common condition and one of the leading causes of vision impairment in people over 50. The National Eye Institute predicts that by 2050, the number of people with AMD in the United States will double, increasing from 2.07 million to 5.44 million.1 AMD destroys the macula, the small region in the middle of the retina responsible for central vision, thereby reducing one’s ability to see objects in the center of their field of vision. There are three stages of AMD—early, intermediate, and late—with late-stage disease further classified into geographic atrophy (or dry) AMD and neovascular (or wet) AMD, both of which produce vision loss. Wet AMD is associated with the growth of new, abnormal blood vessels in the retina that leak fluid, causing inflammation and further damage.2 It can produce rapid and severe vision loss.

DME is a serious complication of diabetes caused by a buildup of fluid in the macula resulting from leaking blood vessels. DME can also lead to vision loss or even blindness over time. Driven by rising rates of diabetes, the number of cases of diabetic retinopathy in the United States is expected to nearly double by 2050, rising from 7.7 million to 14.6 million.1 Approximately half of all people with diabetic retinopathy will develop DME.2

Addressing the Need

Only two drug classes, anti-vascular endothelial growth factor (anti-VEGF) and corticosteroids, are currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of wet age-related macular degeneration (wAMD) and DME. While effective, these therapies do not fully address the complex pathology that leads to progression of these retinal diseases.

Available intravitreal steroid implants used to treat DME provide a duration of activity of either a few months or several years. Retinal specialists report that for many patients, a more appropriate duration of therapy would be six months. This treatment interval could allow clinicians to reduce injection frequency while permitting adequate control over and monitoring of efficacy and adverse events.

Current anti-VEGF agents require frequent injections (as often as every 4 – 6 weeks) to maintain visual gains, which create a significant burden for patients, physicians, and the entire healthcare system. They may also lose efficacy over time. In this situation, the only option currently available is to switch to another agent in the same class.

The current anti-VEGF therapies are large proteins, monoclonal antibodies or decoy receptors, that target one or two of the four VEGFs. Small molecule drugs that block all VEGF signaling by inhibiting all three VEGF receptors could potentially provide greater efficacy but have a very short half-life in the eye. Without some form of sustained release technology, small molecule drugs would have to be injected too frequently to make them practical. Therefore, to realize the potential promise of small molecule therapeutics for retinal diseases, the challenge of delivering them to the back of the eye must be overcome.


  1. National Eye Institute. Age-Related Macular Degeneration (AMD). National Institutes of Health. Accessed January 9, 2018.
  2. National Eye Institute. Facts About Age-Related Macular Degeneration. National Institutes of Health. Accessed January 9, 2018.​