Macular Degeneration Therapeutic Program
We have identified several miRNAs whose expression levels are altered in patient samples as well as animal models of the disease. Animal experiments are planned to evaluate the therapeutic efficacy of introducing single and combinations of synthetic miRNAs into the eyes of animal models.
Age-related macular degeneration (ARMD) is a degenerative condition of the macula (the central retina). It is the most common cause of vision loss in the United States in age 50 or older, and its prevalence increases with age. The photoreceptors and the retinal pigment epithelium (RPE) rely on each other for functionality, and the death of one layer leads to the death of its partnered layer. The degeneration of the cells leads to drusen formation between the choroid and the RPE layer. This deprives the sensitive retinal tissue of oxygen and nutrients that it needs to function and thrive. As a result, the central vision deteriorates. Macular degeneration may be caused by variety of factors: genetics, age, nutrition, smoking, and sunlight exposure may all play a role.
Macular degeneration is a progressive eye condition affecting as many as 15 million Americans and millions more around the world. There are several types of macular degeneration, but the fastest growing form is age-related macular degeneration (AMD). AMD is the number one cause of vision loss and legal blindness in adults over 60 in the U.S. As our population ages and the "baby boomers" advance into their 50's and 60's, we will see a virtual epidemic of AMD. Perhaps 14%-24% of the U.S. population aged 65-74 years and 35% of people aged 75 years or more have the disease.
Although there are no FDA-approved drugs for the treatment of macular degeneration, late stage clinical trials are ongoing for several candidates. Among these are two oligo-based therapies. One is an aptamer that inhibits the activity of vascular endothelial growth factor (VEGF); the second is an siRNA that targets the expression of the VEGF receptor. Both compounds have displayed clinical efficacy, revealing the potential for using oligo-based therapies in the treatment of diseases of the eye.