Allergan adds RP gene therapy program to project pipeline

According to an Allergan statent, RST-001 is a “first-in-class gene therapy application of optogenetics, a therapeutic approach that confers light sensitivity to cells that were not previously, or natively, light sensitive”.{{quote-A:R-W:450-Q: RST-001 is a first-in-class gene therapy application of optogenetics }}“By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, the technology introduces additional light sensitivity to the retina,” the statent explained.“The RST-001 optogenetic approach ploys a photosensitivity gene, channelrhodopsin-2, to create new photosensors in retinal ganglion cells to potentially restore vision in retinal degenerative conditions.”In August 2015, RetroSense’s investigational new drug application for RST-001 received clearance from the US FDA.{{image2-a:r-w:400}}In March this year, RetroSense initiated a phase I/IIa clinical trial to evaluate the safety of RST-001 in patients being dosed, and in August, it was said the low dose cohort of patients had been safely dosed.Allergan chief research and development officer Mr David Nicholson said the RST-001 program could be “a real breakthrough in the treatment of unmet needs across a host of retinal conditions, including RP” and that Allergan was “excited by the prospect of advancing an entirely new approach in the treatment of retinal diseases”.