Retinal diseases (ReN003)

Retinal degenerative diseases such as retinitis pigmentosa ('RP') and age-related macular degeneration ('AMD') are a major cause of blindness in the industrialised world. The use of stem cell technology in such diseases is especially attractive as it provides the ability not only to halt disease progression but also to reverse the loss of function through replacement of lost photoreceptors and accessory cells.

In conjunction with our academic partners, including the Schepens Eye Research Institute at Harvard Medical School, we are pursuing a development programme, ReN003, to develop novel retinal stem cell lines to clinical standard as a potential cell therapy treatment for degenerative diseases of the retina.

Market opportunity

Patients blinded through disease have become one of the largest areas of unmet medical need; there are more than 50 million patients in the US alone.

AMD is the main cause of blindness in the elderly in the Western world, with 30 per cent of the over-seventies suffering. Worldwide incidence is estimated at 25 million patients. RP is one of the most common inherited causes of blindness in people aged between 20 and 60, affecting 1.6 million people worldwide.

There are currently no established treatments for retinal degenerative diseases, with existing therapies focused on preventing further degeneration using antioxidants or vitamins. Experimental cell and gene therapies have been in existence for some time, however. Eye surgery is commonplace today, and surgeons in the field believe that cell transplantation therapies should be no more difficult than more conventional surgical treatments.

Progress

Our academic partners have already demonstrated the viability of this programme in early experimental work on a separately sourced cell line. We are now deriving our own human retinal cells to GLP standard ahead of pre-clinical efficacy testing.

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