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Pioneering surgeon completes world’s first AMD gene therapy

05/03/2019By Myles Hume
A University of Oxford professor who conducted the first gene therapy trials in two rare ocular diseases has now completed the same feat for age-related macular degeneration (AMD).

Professor Robert MacLaren completed the landmark procedure on 80-year-old British woman Mrs Janet Osborne, of Oxford, in a clinical trial at the John Radcliffe Hospital, offering hope for patients with early-stage AMD.

According to MacLaren, the operation involved detaching the retina and injecting a solution with a virus containing a modified DNA sequence. The virus is designed to infect the retinal pigment epithelium (RPE) cells, and correct the genetic defect causing the disease.

“When the virus opens up inside the retinal cell it releases the DNA of the gene we have cloned, and the cell starts making a protein that we think can modify the disease, correcting the imbalance of the inflammation caused by the complement system,” MacLaren said.

Prof Robert MacLaren
Prof Robert MacLaren
“This is a rapidly evolving field. Given that we understand a lot more now about the manufacture of the treatment, and the effects of the virus when doing gene therapy at the back of the eye.”
Robert MacLaren, University of Oxford

“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, and we hope that in future it will slow down the progression of macular degeneration.”

It is hoped the therapy will halt progress of the condition and preserve what vision patients have remaining. If successful, the procedure could be particularly useful for patients diagnosed with early-stage AMD.

Last week’s procedure is the third time MacLaren has successfully conducted a world-first gene therapy procedure after he completed trials in patients with choroideremia in 2011 and retinitis pigmentosa in 2017.

“This is a rapidly evolving field. Given that we understand a lot more now about the manufacture of the treatment, and the effects of the virus when doing gene therapy at the back of the eye, as well as all the other gene therapy programmes being developed at the moment, I would hope that we’ll see a treatment for people with dry AMD within the next few years,” he said.

The latest operation was part of the FOCUS trial, which is sponsored by UK biotech company Gyroscope Therapeutics. The company develops gene therapy products for ocular diseases.

Some of the enabling viral vector development also occurred at the University of Oxford.

Designs for Vision

Sir Peter Lachman, the scientist from the University of Cambridge who led the work on the complement system leading to the creation of Gyroscope Therapeutics, said: “We have a better understanding now on the relationship between the complement system and the AMD disease which lead us to the discovery that restoring the balance of a hyperactive complement system could be a potential therapeutic approach in dry AMD.”


More reading:

Gene therapy for incurable disease improves vision after 5 years
Gene therapy shows promise for reversing blindness
'World first' robotic eye sugery successful


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