RetroSense Gets Intellectual Property Deal

ANN ARBOR — The Ann Arbor biopharmaceutical startup RetroSense Therapeutics announced a license agreement for exclusive, worldwide rights to Massachusetts General Hospital’s patent application entitled “Method for Augmenting Vision in Persons Suffering from Photoreceptor Cell Degeneration.”

The licensed intellectual property is based on methods for restoring or improving vision using optogenetic approaches. The patent is based on research conducted by Richard Masland, Ph.D., director of the Cellular Neurobiology Laboratory in the MGH Department of Neurosurgery.

“Licensing of this patent application is a key component of our intellectual property development efforts, as we continue to seek ways to expand and further defend our position,” said RetroSense CEO Sean Ainsworth.

RetroSense is also working on a compound called RST-001, which is close to clinical trials, Ainsworth said. Recently designated with Orphan Drug Status, RST-001 is being developed as a first-in-class gene therapy application of optogenetics designed to restore vision to those affected by retinal degenerative conditions.

Optogenetics refers to 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 conferring 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, no matter what gene or mutation has caused it.

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.

Said MGH’s Masland: “Much of the research from our lab was conducted to investigate new options for patients suffering from retinal disease. We are encouraged by the progress made at RetroSense and recognize the importance of the orphan drug designation which should further enable development of novel treatment options for patients with severe vision loss due to retinitis pigmentosa.”

RetroSense is working on several gene therapies designed to restore vision in patients suffering from blindness due to retinitis pigmentosa and advanced dry age-related macular degeneration (advanced dry-AMD). 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.

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