Acucela to develop retinal gene therapy

Acucela Inc. announced that it will develop the University of Manchester’s human rhodopsin based optogenic gene therapy for the treatment of degenerative retinal diseases, including retinitis pigmentosa. The company expects the treatment will restore some level of vision in patients who are legally blind. 

The therapy seeks to offset the decline of photoreceptor cells caused by genetic mutations by stimulating retinal neurons to develop light-sensitive properties. Using a viral vector, the signaling cells that connect photoreceptors to optic nerve ganglia will be transduced to produce rhodopsin, the receptor protein found in rod cells.

Investigators at the University of Manchester have shown the process can restore visual response in a mouse model of retinal degeneration. According to the press release, rhodopsin’s biological mechanism imparts a relatively high level of light sensitivity for this type of therapy and as a human protein, may minimize any immunological impact.

“Having observed a dramatic recovery in vision in a mouse model of RP, we are delighted at the prospect of working with Acucela towards restoring useful vision in patients with advanced hereditary retinal degenerations,” stated Dr. Paul Bishop, FRCOphth, PhD, Professor of Ophthalmology, University of Manchester.