VIDEO: Rare Eye Disease

Ophthalmology orphan diseases

MANF continues to perform on so many different levels for Amarantus. While the recent focus has been on the Company’s nearer-term product candidate, it is critically important to remember the vast potential of our program. What I find most remarkable about the sheer breadth of the Company’s lead therapeutic program is that, in the end, all of its potential applications to treat diseases in various organs throughout the body, including the brain, heart, and most recently the eye, are based on the exact same fundamental biological mechanism: mitigating .

(RP) is a chronic progressive degenerative disorder of the eye that is characterized by the death of photoreceptor cones and rods in the retinal pigment epithelium (RPE). It is a genetic disease that affects roughly 100, 000 people in the United States, 100, 000 people in Europe and 50, 000 people in Japan. Due to its relatively small patient population, RP qualifies as an orphan indication which provides a number of financial incentives for development of MANF in this indication. Further, there is a very significant unmet medical need as few therapies effectively treat this population of patients, leaving most legally blind before they reach the age of 50. Moreover, delivery of MANF directly into the eye is far simpler than Convection Enhanced Delivery in Parkinson’s, or systemic delivery in Ischemic Heart Disease. Together, the components of a smaller patient population, a significant unmet medical and a local delivery need may provide an ideal opportunity for the rapid clinical development of MANF that could lead to commercialization years ahead of larger indications for MANF.

We believe RP is just the beginning of the MANF story in ophthalmology. Other neurotrophic factors have begun to show promise in many diseases in the area and the announced earlier this week scientifically supports development in many therapeutic indications beyond RP in ophthalmology, including Dry Age-Related Macular Degeneration (Dry AMD). Dry AMD affects approximately 15 million people in the United States. 14%-24% of the U.S. population age 65-74 years and 35% of people aged 75 years or older have the disease[1]. Currently, there are no available disease-modifying treatments for Dry AMD, making the opportunity for MANF so attractive. MANF’s development in RP may accelerate the pathway for it to reach the significantly larger Dry AMD patient population by proving cone and rod protection in humans in the smaller RP population first.

Popular Q&A

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Does the center for disease control and prevention conduct research in epidemiology?

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