Oxford Biomedica and Santen Collaborate on Gene Therapy Vectors for Retinal Disease

Oxford Biomedica has entered into an R&D collaboration and option and license agreement with specialty pharma Santen Pharmaceutical to research and develop gene therapy products for the treatment of an inherited retinal disease.

Inherited retinal diseases are a group of rare disorders caused by one of more than 260 different genes, where mutation results in vision loss or blindness, often disproportionally affecting children and young adults with few or no therapy options.

These conditions are ideal candidates for gene therapy because many of the responsible genetic mutations have already been identified. In addition, the eye is a readily accessible organ conducive to direct delivery of gene therapy vectors to the diseased tissue. A key advantage with lentiviral vectors is their ability to deliver large therapeutic genes, which is challenging with other vector systems.

The deal with Santen marks Oxford Biomedica’s first foray into Japan.

Under the terms of their agreement, the collaboration will generate preclinical proof of concept of an inherited retinal disease gene therapy using lentiviral vectors developed and manufactured by Oxford Biomedica. The collaboration includes a license to use Oxford Biomedica’s LentiVector platform and access to its industrial-scale manufacturing capabilities. Oxford Biomedica is entitled to an undisclosed milestone payment on exercise of the option to the LentiVector platform as well as development milestones, and up to a 10 percent royalty on net sales. Santen has worldwide commercial rights to the program, while Oxford Biomedica retains an option to co-fund and participate in development and commercialization in the United States and Europe.

“A gene therapy approach to treating inherited retinal diseases will allow Santen to meet the needs of patients suffering from inherited retinal dystrophies,” said Naveed Shams, chief scientific officer and head of global R&D at Santen. “This important collaboration builds on Santen’s ongoing research efforts as part of the CiCLE Programme from the Japan Agency for Medical Research and Development, and further strengthens our commitment to addressing challenges in ophthalmic care.”

Photo: Naveed Shams, chief scientific officer and head of global R&D at Santen

Author: Rare Daily Staff