Blindness breakthrough: UK firm REVERSES vision loss in ‘revolutionary’ gene therapy study | Science | News

Revolutionary gene therapy could help cure serious illnesses

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

The large majority of work undertaken by MeiraGTx’s researchers focuses on eye programs for inherited retinal diseases.

Inherited diseases, Dr Forbes explained, are those where someone has a broken or missing gene that is causing problems — and gene therapy allows experts to insert a replacement.

MeiraGTx’s key breakthrough lies in the development of “second generation” gene therapies in which the inserted gene will be able to be switched on and off as desired with a pill.

The firm’s most advanced line of research to date involves an ongoing multi-phase trial around an inherited retinal disease that has recruited patients from the UK, Ireland, Europe and the US.

The researchers have shown that their therapy 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 would otherwise end up blind.

Gene therapy and an eyeball

Gene therapies developed by a UK firm can reverse progressive vision loss (Image: Getty Images)

Zandy Forbes

MeiraGTx CEO Dr Zandy Forbes told Science Digest: ‘We use genes as actual drugs’ (Image: Science Digest)

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

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

However, she added, “the patients that we’ve treated, some of them were able to do things like navigate through a maze more rapidly, they reported that they could see better in dark conditions.

“And when we measured the sensitivity of their retinas, they appeared to have an increase.

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

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Alongside addressing inherited diseases, Dr Forbes said that their gene therapies can also be used to treat other serious diseases that are not genetic in nature.

She explained: “We have a program in Parkinson’s disease, for example, where we’re able to put a tiny dose of a specific gene therapy into the tiny part of the brain which controls movement — and goes wrong in Parkinson’s.

“In doing that, we can rewire that part of the brain and we’ve shown that we help patients with their movement defects in Parkinson’s.

“It allows us to change the signaling in the brain in a way that allows movement to occur more normally.”

Other diseases that MeiraGTx’s 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 (Image: Creative Commons / Christian Hamel)

The MeiraGTx lab in Shoreditch

Pictured: The MeiraGTx lab in Shoreditch (Image: Science Digest)

Put together, Dr Forbes notes, the various gene therapies have the potential to significantly improve human quality of life and longevity.

She explained: “What I just said about infectious diseases, degenerative diseases and diseases of metabolism — by addressing those three really large health problems in the world, we should be able to increase people’s life in a way that is not sci-fi.

“Those are big problems, and [tackling] just those will enhance people’s longevity, as well as their quality of life and their health, for however long they live.

“Really, we are looking at the diseases that affect us today, and in addressing those we hope to increase the health and longevity of the population in general.”

An infographic on Parkinson's disease

Dr Forbes said: ‘We have a program in Parkinson’s disease’ (Image:

What sets MeiraGTx apart from its competitors, Dr Forbes said, is that they not only have the laboratories that test the new gene therapies, but at their site in Shoreditch they have the labs which have researched and invented a way of manufacturing those gene therapies.

The London-based center also includes a gene therapy manufacturing facility which is approved to produce therapies for use in clinical trials.

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

“And that’s actually helped us be much more efficient in our development of these products and also in our ability to communicate with regulatory agencies who then are the groups that allow you to use the products in people and approve them eventually.”

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