Axordia

Treating Age-related Macular Degeneration

Age-related Macular Degeneration (AMD) is the world’s largest cause of blindness. It exists in two forms, wet and dry, of which the dry version accounts for about 80% of the market. The dry form is characterised by the slow breakdown of the light sensitive cells in the macula, in particular the loss of retinal pigment epithelium (RPE), resulting in gradually blurring central vision in the affected eye and a mild loss of peripheral vision.

The disease is hereditary and because it is related to age is becoming a major public health problem:

• one third of Americans over the age of 75 have some form of AMD;
• in the USA the NIH estimate that the number of AMD sufferers is set to rise from 1.8 million to 2.9 million by 2020; and
• in the UK alone up to half of those 30,000 people per annum who are registered as blind or partially sighted have the dry form of the disease. The long term health care costs for these patients are significant and the effect of this disease on the patient’s quality of life is marked

Currently there is no approved treatment for the dry form of the disease.

Work in animal models is underway to demonstrate the safety and effectiveness of Axordia RPE cells in treating AMD and early data is encouraging.

RPE cells can be sourced from organ donor material, but this is difficult to obtain and the RPE cells show a limited capacity to grow in culture.

Notably Axordia can derive human RPE cells from human embryonic stem cells. Human embryonic stem cells commonly sourced from surplus material supplied by IVF programmes readily proliferate in culture providing a renewable raw material to generate RPE cells.

Axordia is developing a therapeutic treatment for AMD using RPE cells derived from Axordia’s HESC GMP lines.

Over the next two years the project will:

• establish scaleable GMP compliant HESC lines capable of differentiation into RPE cells;
• develop a GMP compatible process to generate RPE cells; and
• produce a pre clinical data set in animals to demonstrate efficacy and safety of the candidate therapeutic cells.

This will lead to Phase I/II clinical trials in a small number of patients in approximately two years.