Oxford Biomedica and Santen have announced plans to enter into a research and development collaboration and option & licence agreement for development of gene therapy vectors for inherited retinal disease.

The aim of the collaboration is to generate pre-clinical proof of concept to treat an inherited retinal disease with lentiviral vectors developed and manufactured by Oxford Biomedica, and includes a licence to use the company’s LentiVector platform.

John Dawson, chief executive officer of Oxford Biomedica, said that the company is “delighted to have formed our first collaboration in Japan. Santen is a leading, multi-national ophthalmology company developing an innovative gene therapy product for the treatment of a significant inherited retinal disease affecting patients with few or no therapy options.

Inherited retinal diseases 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 technically challenging with other vector systems.

John Dawson continued, “We believe that our LentiVector platform is particularly well-suited to the delivery of large genes to the eye. We look forward to working together with Santen in a true parternship that will bring to bear our respective capabilities in gene therapy vectors and inherited retinal diseases to maximise the value of this important R&D programme.”

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.