By Cyndi Root RetroSense Therapeutics announced in a press release that the Food and Drug Administration (FDA) has granted Orphan Drug status to RST-001. The treatment for retinitis pigmentosa (RP) combines gene therapy and optogenetics. RetroSense developed the proprietary technology from research conducted at Wayne State University and Massachusetts General Hospital. Sean Ainsworth, RetroSense CEO, said, 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. Optogenetics Retinitis pigmentosa causes the degeneration and loss of rod and cone photoreceptors in the retina, causing severe vision loss and blindness. Currently there are no FDA-approved drugs to treat RP. RetroSenses work in optogenetics involves making the retina more light sensitive, thereby improving vision. The company expects RST-100 to have broad applications and to be useful in heredity or acquired RP. RST-001 uses a photosensitivity gene, channelrhodopsin-2, and creates new photosensors in the retinal cells. Channelrhodopsin-2 has been shown in numerous animal studies to restore light perception and vision, and in primate studies, the agent was well tolerated. RetroSense is using optogenetics and channelrhodopsin-2 … Continue reading
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