Reference: PD06050
The Problem
Age-related Macular Degeneration (AMD) affects millions of people, progressively robbing them of their central vision and significantly reducing their quality of life. Without central vision, they are unable to read, recognize faces, drive, or perform countless daily activities. The vast majority of AMD cases consist of the “dry” form of the disease, in which the retinal pigment epithelium (RPE) atrophies, leading to the loss of the associated photoreceptor cells. There are no currently approved treatments to halt or reverse the loss of vision once dry AMD is recognized.
The Technology Solution
Researchers in the Department of Ophthalmology at the University of Tennessee Health Science Center have previously demonstrated that certain glycans support outer segment assembly and promote photoreceptor cell survival, even in the complete absence of an RPE. This activity is receptor-mediated and unrelated to the metabolic value of these sugars, as some non-metabolizable sugars showed potent activity. Most potent of all were certain multivalent oligosaccharides, especially asialo triantennary glycan (NA3). NA3 was shown to be effective in Xenopus laevis frogs at an extremely low dose of 1nm. Initial safety studies in NZW rabbits show a safety profile at concentrations of 1 nm and 100nm for a time period up to 14 days. The researchers are continuing to test these compounds in animal models of AMD. They are also currently investigating and developing a nanoparticle delivery system for these sugars.
Related Publications:
Neuron Glia Biol. 2005 Jan;1:1-6;
Mol Vis. 2003 Dec 16;9:701-9
Benefits
• Be first to market with a treatment for dry AMD, which has no FDA-approved treatment options.
• Treatment market expected to be early and late cases of dry AMD.
• Bypass formulation difficulties by developing these compounds which have high aqueous solubility.
• Decreased safety risks due to expected absence of toxic metabolites.
Patents
• US Utility Patent No. 8,092,825. Issued January 2012.
The Inventor
Dr. Monica Jablonski is an Associate Professor of Ophthalmology at the University of Tennessee Health Science Center. Her research interests are mechanisms that regulate photoreceptor outer segment assembly, mouse models of eye disease, proteomics, retinal cell biology, and mutagenesis.