Japanese-US gene therapy company Acucela Inc., has annunced the execution of a licensing deal with the University of Manachester, UK. The exclusive deal will provide commercial access for Acucela to a human rhodopsin based optogenetic gene therapy for the treatment of retinal degenerative disease, particularly rhodopsin related retinitis pigmentosa. The mutation-independent optogenetic therapy aims to transduce ON-bipolar cells with a human rhodopsin transcript under the control of a cell-specific promoter. The principal of expressing functional light activated molecules outside their normal rod and cone cells has been demonstrated in both cell and animal models. The ability to express such molecules in the absence of rods and cones may present significant therapeutic opportunities for patients who have undergone considerable photoreceptor degeneration but maintain other retinal cell types capable of viral transduction.
A research paper in the journal Current Biology (August 2015) outlined the basic technology in which ectopically expressed human rod opsin, engineered to be driven by either a non-selective or ON-bipolar cell-specific promoter, was shown to be capable of physiological functioning despite being located outside the natural photoreceptor host. The expressed rhodopsin was additionally shown to restore visual function in an animal model of advanced retinal degeneration, paving the way for the design of more advanced testing and potential clinical studies. Electrophysiological analysis of animal models harbouring the optogenetic transcripts showed changes in firing induced by light pulses and increases in environmental luminance. Most interestingly, these physiological responses could be induced at light intensities below the normal physiological range of photoreceptors.
Commenting on the licensing transaction, Dr. Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela stated, “We are extremely excited to enter into this collaboration with the University of Manchester and to begin the important development work needed to unlock the potential of optogenetic gene therapy to improve visual function in patients who have lost much of their vision as well as their hope”. Similarly, Dr. Rich Ferrie, Director of Operations at the University of Manchester Intellectual Property (UMIP) commented, “We are delighted to have entered into this license with Acucela, who we believe is the ideal partner to develop a gene therapy for RP based on the ground-breaking science of Professors Lucas and Bishop. The signing of this agreement represents a potentially pivotal moment and offers real hope for RP patients.”