Researchers at Oxford University are developing an amazing gene therapy technique using cloned DNA may bring relief to patients suffering from macular degeneration.

A key factor in AMD is the complement system, a system of proteins in our immune system that fights bacteria. In macular degeneration, these proteins are over-active and start to attack the retinal cells, in a similar way to how they would attack bacteria.

A developing frontier in gene therapy is ongoing at the Nuffield Department of Clinical Neurosciences (Medical Science Division) at Oxford University.  Lead researchers explain that their team is attempting to  harness the power of the virus, a naturally occurring organism, to deliver the intervention DNA into the patient’s cells. When the virus opens up inside the retinal cell, it releases the DNA of the gene the Nuffield team has cloned, and the cell starts making a protein that is believed to then modify the disease, correcting the imbalance of the inflammation caused by the complement system.

The idea of this gene therapy is to ‘deactivate’ the complement system, but at a very specific point at the back of the eye, so the patient would otherwise be unaffected by it.  Researchers hope that in future it will slow down the progression of macular degeneration, and actually halt the progress of the condition and preserve what vision patients have remaining. If successful, it is hoped that gene therapy can be used in the future on patients with early AMD and so halt the disease before their vision has started to deteriorate.

Gyroscope Therapeutics Holdings plc (“Gyroscope”), the commercial holding entity for the gene research and development,  was acquired by Novartis, a leading global medicines company in 18 February 2922. The acquisition included  an upfront payment of $800 million (£587 million) and up to $700 million (£514 million) potentially due upon the achievement of certain milestones.