Blindness breakthrough: British company reverses vision loss in ‘revolutionary’ gene therapy study | science | News

Revolutionary gene therapy could help treat serious diseases

MeiraGTx is a London-based company that uses gene therapy to “turn back the clock” on serious diseases. MeiraGTx CEO Dr. Zande Forbes said Science Digest: “We use genes — so, the messages that make the body make proteins — as actual drugs.” “It’s revolutionary because it actually allows your body to produce the drug itself, based on that signal.”

The vast majority of work done by MeiraGTx researchers focuses on eye programs for genetic retinal diseases.

Inherited diseases are those in which someone has a broken or missing gene that causes problems – and gene therapy allows experts to introduce a replacement, Dr. Forbes explained.

The major breakthrough by MeiraGTx lies in the development of “second generation” gene therapies in which the inserted gene can be turned on and off at will using birth control pills.

The company’s most advanced line of research to date includes an ongoing, multi-stage trial on hereditary retinal disease that has recruited patients from the UK, Ireland, Europe and the US.

Researchers have shown that their treatment for the condition – known as X-linked retinitis pigmentosa – is not only safe and well-tolerated, but can also improve the vision of patients who may end up blind.

Gene therapy and the eyeball

Gene therapies developed by a British company could reverse progressive vision loss Image credit: Getty Images

Zandy Forbes

‘We use genes as actual drugs,’ MeiraGTx CEO Dr Zande Forbes told Science Digest. (Photo: Science Digest)

Dr. Forbes said: “Men [in the study] They were treated in one eye, and what we showed is that in various aspects of their vision – particularly in dim light, there appears to be an improvement in their ability to see.

“One of the biggest problems with this disease is […] is that early in the disease, as people start to go blind, they can’t see in the dark. It gets progressively worse and they eventually go blind.”

However, she added, “The patients we treated, some of whom were able to do things like navigate a maze more quickly, and reported that they could see better in dark conditions.

“And when we measured the sensitivity of the retina, they seemed to have an increase.

“All of these things together suggest that replacing this gene has a benefit for those patients who have lost it.”

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Besides treating genetic diseases, Dr. Forbes said their gene therapies could also be used to treat other serious diseases that are not genetic in nature.

She explained: “We have a program for Parkinson’s disease, for example, where we can put a small dose of a specific gene therapy into the small part of the brain that controls movement – and it goes wrong in Parkinson’s disease.

“By doing this, we can rewire this part of the brain and have shown that we are helping patients with motor defects in Parkinson’s disease.

“It allows us to change the signals in the brain in a way that allows movement to occur naturally.”

Other diseases that MeiraGTx researchers are working to treat with their genetic tools include amyotrophic lateral sclerosis (ALS) and diabetes.

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An eye with mid-stage retinitis pigmentosa

Pictured: an eye with mid-stage retinitis pigmentosa (Photo: Creative Commons/Christian Hamel)

MeiraGTx Laboratory in Shoreditch

Pictured: MeiraGTx Lab in Shoreditch (Photo: Science Digest)

Dr. Forbes notes that various gene therapies have the potential to dramatically improve a person’s quality of life and longevity.

She explained, “What I just said about infectious diseases, degenerative diseases, and metabolic diseases – by tackling these three really big health problems in the world, we should be able to increase people’s lives in a way that isn’t science fiction.

“These are big problems, and [tackling] Only those will enhance people’s longevity, as well as their quality of life and health, no matter how long they live.

“Really, we’re looking at the diseases that plague us today, and in tackling those diseases we hope to increase the health and longevity of the population in general.”

Parkinson's disease diagram

‘We have a program for Parkinson’s disease,’ Dr. Forbes said. (Photo:

What sets MeiraGTx apart from its competitors, Dr. Forbes said, is that not only do they have labs that test new gene therapies, but on their site in Shoreditch they have labs that have researched and devised a way to manufacture those gene therapies.

The London-based center also includes a gene therapy manufacturing facility that has been approved to produce treatments for use in clinical trials.

Dr. Forbes added: “So, we go all the way from discovery, research, working out how to manufacture, to actual certified manufacturing – all in this close area.

“It actually helps us to be more efficient in our development of these products and also in our ability to communicate with the regulatory bodies that are then the groups that allow you to use the products on people and eventually approve them.”

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