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news.US-based RetroSense Therapeutics has received orphan drug designation from the Food and Drug Administration (FDA) for its lead product RST-001 to treat retinitis pigmentosa (RP), a genetic condition which leads to the progressive degeneration of rod and cone photoreceptors.
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RST-001 is being developed as a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by RP.
Optogenetics refers to means of conferring light sensitivity to cells that were not previously, or natively light sensitive.
The company is conferring new light sensitivity to the retina, with the expectation of improved or restored vision, by applying optogenetics to retinas in which rod and cone photoreceptors have degenerated.
The approach to using optogenetics in vision restoration is based on research carried out at Wayne State University’s Kresge Eye Institute and Department of Anatomy and Cell Biology, and Massachusetts General Hospital.
The company said that it has worldwide exclusive rights to the relevant intellectual property from both institutions.
RetroSense Therapeutics CEO Sean Ainsworth said: "We are pleased that the FDA has granted Orphan Drug status to our lead product, RST-001.
"This significant milestone will enable us to continue to develop new and innovative treatments for retinitis pigmentosa, a truly debilitating condition.
"We are hopeful that the benefits associated with Orphan Drug status will better enable us to advance RST-001 through development and ultimately into the marketplace where it may benefit many who are suffering from blindness due to retinitis pigmentosa."