Treatment of AMD by Targeting Lipocuscin
Age-related macular degeneration affects about 14 million people in the US and is expanding with the aging of the population. In this disease, daytime vision (cone dominated vision) degrades with time because cone photoreceptors, which are concentrated in the foveal region of the retina, die. Despite the fact that current treatment of AMD are targeting the wet form of AMD, accounting for only about 10% of AMD cases, the market for therapeutics is growing rapidly toward annual sales of $1 billion. Therefore, enabling treatment of the dry form of AMD by Dr. Rando’s approach presents an opportunity not only to relieve an extremely large patient population of a debilitating clinical condition, but also create a commercially attractive multi-billion dollar market.
Therapeutics for the treatment of AMD and Stargadt disease.
Innovations and Advantages
The invention encompasses a new approach for the treatment of macular degeneration, in particular to dry form of age-related macular degeneration (AMD). It consists of a series of small molecule compounds that eliminate the accumulation of retinotoxic Lipofuscin, a byproduct of the visual process. The accumulation of Lipofuscin is known to cause dry AMD. Rando’s approach enables Lipofuscin elimination by limiting the turnover of the visual cycle. Known inhibitors of the visual cycle are mostly toxic retinoids. Rando has identified multiple targets in the visual cycle as well as a series of non-retinoid compounds which are anticipated not to share this toxicity. Key targets and the efficacy of several prototypical compounds have been validated in vitro and in animals with no apparent toxicity.
Availability & Patent Status
Patents have been broadly filed for compositions of matter as well as methods of use of a series on non-retinoid compounds, including the generic structure of fenretinide, in treating ocular disease. The technology is available for international licensing on an exclusive basis.
Intellectual Property Status: Issued U.S. patent nos.: 7,566,808
Rando, Robert R.
For further information, please contact:
Grant Zimmermann, Director of Business Development
Reference Harvard Case #2252