Blindness therapy comes a step closer following licensing deal with US firm

The therapy was developed by University of Manchester researchers Dr Jasmina Cehajic-Kapetanovic and Professors Robert Lucas and Paul Bishop. In advanced RP the photoreceptor (light-sensitive) cells die off, but other neuronal cells are still present in the retina. In trials using RP affected mice with a complete loss of their photoreceptor cells, the scientists used a gene therapy approach which successfully made these other cells light-responsive. This optogenetic therapy was sufficiently effective at restoring visional responses in the mice to allow them to detect spatial patterns presented using an ordinary flat screen display.

Commenting on the license arrangement Dr. Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela said: “We are extremely excited to enter into this collaboration with the University 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.”

Dr. Paul Bishop, FRCOphth, PhD, Professor of Ophthalmology, University of Manchester added: “This is a very exciting therapeutic approach as the blind mice we treated could see surprisingly well in normal lighting conditions, and we think the approach may be safe as we are putting a normal human retinal protein back into the retina, but in cells that don’t normally make it. We are delighted at the prospect of working with Acucela towards restoring some visual function in patients who have severe visual loss from RP and similar conditions.”

The agreement was negotiated on behalf of the University by its technology transfer office, UMIP. Director of Operations at UMIP, Dr. Rich Ferrie commented, “We believe that Acucela is the ideal partner to develop a gene therapy for RP based on this ground-breaking science. The licensing arrangement has the potential to deliver significant economic return to the University if the clinical trials and commercialisation programme are successful. More importantly the signing of this agreement represents a potentially pivotal moment and offers real hope for millions of RP patients around the world.”

The technology was first reported in Current Biology in June 2015 and in The New Scientist in August 2015 and it was also presented at the ARVO eye research conference in the US in May 2015.

The University of Manchester

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Blindness therapy comes a step closer following licensing deal with US firm