ANN ARBOR, Mich.--(BUSINESS WIRE)--RetroSense Therapeutics LLC, a privately-held biopharmaceutical company, today announced the Company’s Investigational New Drug (IND) application for RST-001 received clearance from the United States Food and Drug Administration (FDA). RetroSense is developing RST-001 for the treatment of retinitis pigmentosa (RP), 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.
“The IND for RST-001 is an important milestone for the company. This brings us one step closer to realizing our ambition of improving vision in those individuals with currently incurable blindness,” said Sean Ainsworth, CEO of RetroSense Therapeutics. “There is great promise for the clinical application of optogenetics and this first human clinical trial should provide key insights into the potential for this therapy to treat diseases affecting the eye or brain.”
Dr. Zhuo-Hua Pan, the inventor of RetroSense Therapeutics’ optogenetic approach added, “My hope from early on was to see our work improve the lives of people with vision defects. It is great to see the approach moving imminently into human clinical studies.”
About RetroSense Therapeutics
RetroSense Therapeutics is a privately-held biotechnology company developing life-enhancing gene therapies designed to restore vision in patients suffering from blindness due to retinitis pigmentosa and advanced dry age-related macular degeneration. There are currently no FDA-approved drugs to improve or restore vision in patients with these retinal degenerative conditions. The Company's approach to using optogenetics in vision restoration is based on pioneering, proprietary research conducted at Wayne State University and Massachusetts General Hospital. RetroSense has worldwide exclusive rights to the relevant intellectual property from both institutions. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic.