Investigational Gene Therapy Designated Orphan Drug for Eye Disorder
RetroSense Therapeutics announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to RST-001 for the treatment of retinitis pigmentosa (RP).
RST-001 is a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by RP. The application of optogenetics to retinas in which rod and cone photoreceptors have degenerated, allows new light sensitivity to the retina, with the expectation of improved or restored vision.
RST-001 is expected to have application to all forms of retinitis pigmentosa, independent of causative gene or mutation.
For more information visit Retro-Sense.com.