The company, Vedere Bio II, is working on optogenetic gene therapies based largely on the work of University of California, Berkeley, neuroscientists Ehud Isacoff and John G. Flannery to restore vision. Their first goal is to find treatment for retinitis pigmentosa (RP). They are hoping their treatment will work for most forms of RP, regardless of the mutation that causes it. Their therapy relies on light-sensitive ion channels or G-protein coupled receptors (GPCRs) from mammals. The cells in the eyes for vision (photoreceptors) rely on GPCRs to start the process. These GPCRs are turned on by a small-molecule photoswitch. For example, 11-cis-retinal is a bent form of the photoswitch. When it absorbs light, it straightens and activates the GPCRs. Vedere Bio II is working on a human GPCR that can bind to a synthetic photoswitch similar to 11-cis-retinal. Researchers believe this photoswitch could treat some forms of vision loss caused by a problem in the pathway that makes and recycles 11-cis-retinal. The treatment they are hoping to create will use adeno-associated viruses (AAVs) to carry the therapeutic gene to the eye through an injection. The hope is that this therapy can work on all kinds of RP and perhaps even other forms of vision loss.
What this means for Usher syndrome: Vision loss in Usher syndrome is a type of RP. This research is hoping to create a therapy that can reduce vision loss in all forms of RP. If the study proves to be successful, this could be a possible treatment to reduce the vision loss caused by RP in Usher syndrome.