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Gene Therapy Offers Hope to Millions of Macular Degeneration Sufferers

scientist developing gene therapy product

Losing your vision is one of the most disheartening developments that may occur in life. While there are many conditions that can affect vision, one of the more common is known as wet macular degeneration, which affects millions of people in the U.S. and is primarily a disease of older age. With macular degeneration, there is a progressive loss of one’s central vision, which can create a checkerboard visual perspective that’s blurred as well. Though age-related macular degeneration treatments do currently exist, they are less than ideal; not only do they require frequent eye injections, but they only partially slow the degradation process. For this reason, many hope that a gene therapy product might offer a better answer. Based on recent trials at 4D Molecular Therapeutics, this might well be the case.

a doctor working on a gene therapy product
A new gene therapy product can rewrite the book on macular degeneration, helping countless sufferers of the disease.

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4D Molecular Therapeutics is among a few companies that are testing a specific gene therapy product for wet macular degeneration. In essence, these products all provide a stimulus for cells to make various chemicals designed to reduce the pathologic process. While a few have promise, recent data released from 4D Molecular Therapeutics actually look the most promising. Why is this important? Because roughly 1.5 million older adults in the U.S. alone are at risk of losing their vision from this disease. Therefore, if a gene therapy product provides a better age-related macular degeneration treatment, that’s great news. As such, it’s worth exploring the results of these preliminary trials in a bit more depth.

A Little About Wet Macular Degeneration

In terms of epidemiology, wet macular degeneration is quite common. It is reported that roughly 20 million adults over age 40 years have the condition, and of these, about 10 percent are at risk for going blind. The basic problem with wet macular degeneration is that fluid and blood leak around the macular of the eye. This, in turn, causes blurring of the vision, especially in central visual fields and can even cause spotty loss of vision. Over time, this progression causes thickening of the retina in more severe cases. For these individuals, vision loss is a real threat, which is why a better age-related macular degeneration treatment is needed.

The underlying cause of wet macular degeneration is one that relates to vascular endothelial growth factor, or VEGF. In some people, VEGF is produced in the eye abnormally, triggering blood vessel growth where it shouldn’t. As new blood vessels grow in these aberrant places, they tend to leak. This is the underlying problem that current an age-related macular degeneration treatment tries to address. But while such treatments do help, they do not halt the process completely, which means visual decline continues. In recent years, biopharma companies have suspected that a gene therapy product might be more effective. This is where the current science involving age-related macular degeneration treatment trials exist.

an older dude having trouble seeing
The fight against macular degeneration might be closer to being won.

The 4D Molecular Therapeutics Trial

The latest results investigating 4D Molecular Therapeutics’ gene therapy product was just released recently. It reflected mid-trial data involving a total of 51 participants with known wet macular degeneration. In the trial, there were three arms that involved high dose treatment, low dose treatment, and treatment as usual. All patients were considered more severe in their disease status based in retinal thickening. Likewise, all averaged about 10 treatment injections over the last year of a traditional age-related macular degeneration treatment. Thus far in the trial, the need for another traditional treatment was reduced by 89% in the high dose treatment group, and 63% in this group didn’t need any additional injections to date. This data expressed 24 weeks of intervention with 4D Molecular Therapeutics gene therapy product. Based on these impressive findings, the share price for 4D Molecular Therapeutics doubled, reaching its highest price to date.

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In terms of its gene therapy product, 4D Molecular Therapeutics uses a virus vector to introduce a transgene product. In essence, the transgene encodes for a chemical called aflibercept, which is what wet macular degeneration patients normally receive via injection. But instead of having this injected, the transgene prompts eye cells themselves to make an anti-VEGF chemical. This chemical inhibits VEGF present, thus addressing the abnormal blood vessel formation and subsequent leakage. Based on the trial to date, it appears the gene therapy product significantly reduces the need for frequent injections. Plus, it’s suspected that it may also reduce drug resistance that affects some people receiving these injections over time. These aspects are why there’s been such excitement about this age-related macular degeneration treatment. And it’s another example of how fast the gene therapy field is evolving.

A New Era of Age-Related Macular Degeneration Treatment

age-related macular degeneration treatment in some genes
Age-related macular degeneration treatment can be battled at the genetic level.

Existing therapies for wet macular degeneration involve primarily two options of care. The first, which is most common, are anti-VEGF injections that suppress blood vessel growth in the eye. These are helpful, but they are short acting in nature. This is why many such patients have to have frequent treatments and may develop drug resistance along the way. The other is less commonly used and is termed photodynamic treatment. This age-related macular degeneration treatment uses a laser to activate a chemical called verteporfin. Once activated, verteporfin breaks down abnormal blood cells to hopefully improve vision. This can be used independently or in combination with anti-VEGF injections. But neither look to be as effective as the gene therapy product offerings being studied at present.

While 4D Molecular Therapeutics’ gene therapy product seems impressive, it does have competition in the field. Adverum is currently testing gene therapies as well for wet macular degeneration. However, there appears to be some safety concerns at present raised by its trials. Also, Regenxbio is similarly testing gene therapies as a new form of age-related macular degeneration treatment. In its case, its administration is not as convenient as that offered by 4D Molecular Therapeutics. Understanding this, it looks like the most favored product to market in this new era of treatment may be 4D Molecular Therapeutics’. In any case, it’s evident new gene therapy options of care for age-related macular degeneration are around the corner. And that’s incredible news for millions of older adults.

 

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