RetroSense Therapeutics’ Investigational New Drug (IND) application for gene therapy based clinical trial for their product RST-001 has received clearance from the US Food and Drug Administration. RetroSense is developing RST-001 for the treatment of retinitis pigmentosa, a genetic condition that leads to the progressive degeneration of rod and cone photoreceptors (cells found in the retina that sense light), resulting in severe vision loss and blindness. With its IND now in effect, RetroSense expects to initiate a Phase I/II clinical trial by year-end in order to evaluate the safety and, potentially, efficacy of RST-001.
RetroSense Therapeutics is developing RST-001 as a first-in-class gene therapy application of optogenetics. Optogenetics refers broadly to means of conferring light sensitivity to cells that were not previously, or natively, light sensitive. By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, RetroSense is working to confer new light sensitivity to the retina, with the expectation of some degree of improved or restored vision for affected patients.
In 2014, the FDA granted Orphan Drug designation for RST-001 based on its development as a treatment of RP, a rare disease that affects an estimated 100,000 people in the United States. As a designated Orphan Drug, RST-001 is eligible for various development incentives under the Orphan Drug Act, including a potential waiver from FDA’s application user fees, certain tax incentives and Orphan Drug exclusivity.
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