Target Disease: Retinal Diseases
The retina is a neuronal tissue located at the back of the eye. Its primary function is the perception of light, the processing of light induced stimuli, and the transmission of light-dependent information to the brain.
Retinal diseases like wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and geographic atrophy (GA) can lead to permanent vision loss. VEGF is a protein produced by cells which stimulates the formation of new abnormal blood vessels, a process called neovascularization, and induces vascular permeability, leading to leakage and swelling of the retina. Swelling of the retina leads to vision decline and death of the retinal cells, which can irreversibly cause blindness if not adequately treated.
In addition, a large number of hereditary diseases affect the retina and can result in severe vision impairment or blindness.
Retinal diseases like wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and geographic atrophy (GA) can lead to permanent vision loss. VEGF is a protein produced by cells which stimulates the formation of new abnormal blood vessels, a process called neovascularization, and induces vascular permeability, leading to leakage and swelling of the retina. Swelling of the retina leads to vision decline and death of the retinal cells, which can irreversibly cause blindness if not adequately treated.
In addition, a large number of hereditary diseases affect the retina and can result in severe vision impairment or blindness.
Geographic Atrophy
About Geographic Atrophy (GA)
Geographic Atrophy is an advanced form of dry age-related macular degeneration (AMD). The pathophysiology of the condition remains unclear and although advances in research have identified several therapeutic targets, there are currently no approved treatments to prevent either the onset or progression of GA.
PREVALENCE OF GA
Geographic Atrophy
42%
OF PATIENTS ARE LEGALLY BLIND WITH A VISUAL ACUITY OF 20/200
GA affects more than five million people globally, and more than 100,000 new cases are diagnosed each year.
GA is responsible for 10-20% of cases of legal blindness and 42% of GA patients are legally blind with a visual acuity of 20/200.
GA is responsible for 10-20% of cases of legal blindness and 42% of GA patients are legally blind with a visual acuity of 20/200.
The Unmet Need of Patients
with GA
Geographic Atrophy is a devastating blinding disease without any approved or effective treatment currently available to prevent its onset or slow down its progression. GA was found to severely impact patients’ ability and confidence to drive, and consequently their independence.
Currently vitamin supplements are recommended for the prevention of the risk of progression of GA, however, the risk can be reduced only by 25%. Low vision aids like magnifiers or special eyeglasses may be prescribed.
Recent clinical trials have indicated that targeting the complement pathway is a valid approach for slowing disease progression, but those investigational therapies require up to 12 injections per year.
Currently vitamin supplements are recommended for the prevention of the risk of progression of GA, however, the risk can be reduced only by 25%. Low vision aids like magnifiers or special eyeglasses may be prescribed.
Recent clinical trials have indicated that targeting the complement pathway is a valid approach for slowing disease progression, but those investigational therapies require up to 12 injections per year.
The Pipeline
GB-701
GB-701 is being developed as a long-acting, locally administered formulation of a potent factor B inhibitor targeting the complement cascade which plays a role in AMD.
Graybug and Insilico Medicine, a clinical-stage, end-to-end artificial intelligence (AI)-drug discovery company, formed a strategic partnership to leverage Insilico’s AI-driven small molecule discovery platform with Graybug’s unique ocular drug delivery technologies enabling Graybug to develop a sustained-release, locally administered ocular formulation of a potent factor B inhibitor as a potential treatment for Geographic Atrophy and intermediate AMD.
Geographic Atrophy is a devastating blinding disease without any approved or effective treatment currently available to prevent its onset or slow down its progression.