Updated on 7 September 2016
Allergan plc and RetroSense Therapeutics LLC, a private, clinical-stage biotechnology company focused on novel gene therapy approaches to restore vision in patients suffering from blindness, announced that Allergan has acquired substantially all of the assets of RetroSense in an all-cash transaction. Under the terms of the transaction, Allergan has paid RetroSense a $60 million upfront payment, and has agreed to potential regulatory and commercialization milestone payments related to its lead development program, RST-001, a novel gene therapy for the potential treatment of Retinitis Pigmentosa (RP).
"The acquisition of RetroSense and its RST-001 program builds on Allergan's deep commitment to eye care, and our focus on investing in game-changing innovation for retinal conditions, including Retinitis Pigmentosa, where patients desperately need treatment options," said Brent Saunders, CEO and President of Allergan.
Retinitis Pigmentosa (RP) is a group of rare, inherited genetic disorders characterized by progressive peripheral vision loss and night vision difficulties followed by eventual central vision loss and blindness in many cases.
RST-001 is 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. By applying optogenetics to retinas in which rod and cone photoreceptors have degenerated, the technology introduces additional light sensitivity to the retina. In 2014, RST-001 received an Orphan Drug Designation by the US FDA for the treatment of Retinitis Pigmentosa.
The RST-001 optogenetic approach employs 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 (IND) application for RST-001 received clearance from the United States Food and Drug Administration (FDA). In March 2016, RetroSense initiated a Phase I/IIa clinical trial to evaluate the safety of RST-001 in patients being dosed, and in August 2016, the low dose cohort of patients had been safely dosed.