Age‐Related Macular Degeneration

Age-related macular degeneration (AMD) is the leading cause of vision loss in Americans aged 60 and older. AMD is characterized by loss of sharp central vision, and can exist in two forms. In dry AMD, the light-sensitive cells in the macula slowly break down, gradually blurring central vision in the affected eye.  In wet AMD, abnormal blood vessels behind the retina start to grow under the macula creating edema and leaking blood into the retina, leading to rapid vision loss. Wet AMD, although less common than dry AMD, results in the majority of the vision loss caused by AMD.

Status

The most effective treatment for wet AMD is intraocular injections of a monoclonal antibody fragment that neutralizes the effects of the angiogenic protein VEGF, resulting in decreased formation of the abnormal blood vessels.  Although effective, this treatment requires frequent injections into the eye to maintain efficacy.  The proposed therapeutic is a one-time injection of ReGenX’s AAV Vector Technology encoding an anti-angiogenic protein in the subretinal space.  Detailed studies have shown that AAV7 is more efficient than other AAV serotypes and is more specific in delivering genes to the target cell type in the retina.  The proposed therapeutic is in the preclinical stage of development.

Market

Wet AMD affects approximately 1.75 million Americans¹. The yearly cost for the medication injected directly into the eye of wet AMD patients is estimated at $24,000 per patient ² ($1.8 billion total for current users), which does not include the costs of the doctors’ visits and assessments of eye structure and function.