Key Enabling Technology for the Development of Novel Therapeutics for Age-Related Macular Degeneration

Summary

• Age related Macular Degeneration (AMD) is estimated to afflict ~196 million people worldwide
• Although people rarely go blind from it, AMD can severely affect central vision and the ability to make out fine details
• Other than vision rehabilitation programs and low-vision devices, there is no real treatment for AMD
• Dr. Sung a key leader in this space is developing more accurate mouse models to study the pathogenesis of AMD and develop novel therapies to treat the disease

Technical Overview

AMD is comprised of two distinct types: Wet and Dry
Wet AMD: Less common, responsible for severe vision loss, clinically manageable

• Caused by abnormal blood vessels growing beneath the retina that leak fluid and blood

Dry AMD: Accounts for >80% of AMD cases

• Light-sensitive cells in the macula slowly break down and form drusen which result in vision loss
• Exact causes of this type is unknown

Dr. Sung and colleagues have developed a new genetically engineered mouse model of Dry AMD by deleting the CLIC4 gene from RPE cells.

Using their novel mouse model, Dr. Sung and colleagues have uncovered that lipids that form drusen derive from the retina lining RPE cells rather than from blood.

Their findings represent a new treatment strategy ripe for further development and application.

Market Opportunity

• Age-related macular degeneration is the most common cause of severe loss of eyesight among people 50 and older
• Understanding disease pathology is crucial for developing novel treatments
• Dr. Sung’s novel mouse model for Dry AMD has uncovered novel pathways and potential treatment targets for AMD
• The team is continuing to use their model to elucidate and advance understanding of dry AMD and eventually test novel therapies

Partnering Opportunity

Weill Cornell Medicine is seeking an industrial partner with deep domain expertise and a presence in the Ophthalmology space to advance this avenue of research and develop novel therapeutics to treat Dry AMD

Figure 1: Normal and AMD Pathology (Source)