RetroSense Therapeutics, LLC, a privately-held biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for the Company’s lead product RST-001 for the treatment of retinitis pigmentosa (RP). Retinitis pigmentosa is a genetic condition which leads to the progressive degeneration of rod and cone photoreceptors (cells found in the retina of the eye that sense light). Loss of these cells results in severe vision loss and blindness. RetroSense Therapeutics is developing RST-001 as a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by RP. By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, RetroSense is conferring new light sensitivity to the retina, with the expectation of improved or restored vision.
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