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Press Release

FDA Gives “Breakthrough” Status to Potential New Treatment for Vision Disease

Early BrightFocus Funding Key to Promising New Development

Ilyas Washington, PhD

Clarksburg MD - Rooted in key, early funding from BrightFocus, a proposed treatment for Stargardt’s disease – an inherited blinding retinal degenerative condition beginning in childhood – has received “Breakthrough Therapy Designation” from the U.S. Food and Drug Administration, allowing for expedited testing and federal review towards possible patient use.

The proposed drug, a chemically modified oral form of Vitamin A, is also being tested for use in age-related macular degeneration (AMD), including an advanced form known as geographic atrophy whose current lack of effective treatment is a major unmet need in vision health. AMD, a non-inherited disease characterized similar to Stargardt’s and marked by a progressive loss of central vision, is the leading form of blindness among older adults, currently impacting the sight of 11 million Americans.

Known in scientific terms as ALK-001 (C20-D3-vitamin A), the proposed therapy is based on foundational work done by Ilyas Washington, PhD, a former Columbia University chemist and co-founder of Alkeus Pharmaceuticals, the developer of this potential drug. BrightFocus awarded Dr. Washington with one of the first research grants of his scientific career, allowing him to begin exploring a possible role for enriched Vitamin A in treating vision disease.

“BrightFocus was the first major funder of my academic lab. They gave me the opportunity – and the confidence – to believe that I could someday stop someone from losing their sight,” Dr. Washington said.

“This is an exciting, hopeful time for vision research. The work of Dr. Washington and his team shows us the power of science to save sight and the critical importance of key, early funding for bold new ideas,” said BrightFocus Foundation President and CEO Stacy Pagos Haller.

This content was first posted on: July 20, 2021

The information provided in this section is a public service of BrightFocus Foundation, and should not in any way substitute for the advice of a qualified healthcare professional, and is not intended to constitute medical advice. Although we take efforts to keep the medical information on our website updated, we cannot guarantee that the information on our website reflects the most up-to-date research. Please consult your physician for personalized medical advice; all medications and supplements should only be taken under medical supervision. BrightFocus Foundation does not endorse any medical product or therapy.

Some of the content in this section is adapted from other sources, which are clearly identified within each individual item of information.

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