Fighting blindness with bugs: can gene infection save people’s sight?


Age-related macular degeneration (AMD) has been designated as a priority eye disease by the World Health Organization.

The disease is the third-ranked cause of blindness globally and an ageing population means that AMD cases are only set to increase, with a projected 196 million predicted by 2020.

One novel treatment has the potential to preserve the vision of those affected by wet AMD, the type characterised by high levels of blood vessel growth within the eye.

Injecting and infecting

Partnered with sponsor Genzyme, Professor Peter Campochiaro has undertaken a preliminary study using an altered common cold virus to deliver reparative genes to the site of AMD.

The virus is injected into the patient’s eyes, where it infects retinal cells and deposits a gene responsible for producing therapeutic protein FLT01. FLT01 acts to halt the growth of leaky blood vessels by suppressing the growth-driving molecule VEGF.

By producing a factory of FLT01-producing cells, these scientists hope to revolutionise the treatment of wet AMD, which currently requires injections of anti-VEGF drugs every six to eight weeks in order to stave off vision loss.

Seeing is believing

More than half of treated patients saw a reduction in fluid within the eye after just one treatment. This prevents scarring and damage to the eye that causes AMD-related blindness.

While the long-term benefits are not completely clear, researchers have predicted that the therapeutic effect of this gene infection could last for years, allowing patients to live an uninterrupted life with AMD.

It also comes with almost no side effects, even in high doses.

Looking ahead

While promising, this treatment has only completed Phase I trials, with a small preliminary group. Therefore, it has a long road ahead before entering the clinic.

However, if successful, this gene therapy could have a huge impact on the market and offer millions of patients the chance to retain their vision.

For more strategic insight into drug development trends, take a look at GlobalData's latest reports.