The First Gene Therapy That Fixes Hereditary Blindness May Finally Get FDA Approval

It would be the first treatment in the U.S. that fixes an inherited genetic trait.

10. 12. 17 / Brad Jones
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Second Sight

Today, a panel will advise the US Food and Drug Administration whether Luxturna, a gene therapy treatment developed by Spark Therapeutics, should be approved for general usage. The treatment has already been used to improve the eyesight of more than two dozen people with retinal disorders.

Gene therapy typically uses an engineered virus to administer a patient with a faulty gene with a corrected version. Rather than simply responding to the symptoms of the condition in question, it attempts to make changes to the individual’s genetic make-up in order to solve the problem at its root.

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Luxturna fixes a mutation in a gene known as RPE65, which is responsible for telling the body how to produce a protein that’s essential for normal eyesight. It introduces billions of engineered virus particles bearing a corrected version of the gene to the retinal cell, via a quick injection to the eyes.

The company estimates that 6,000 people around the world could benefit from Luxturna, including between 1,000 and 2,000 people in the US who suffer from diminished eyesight. The majority of these people would eventually lose their current level of vision entirely without treatment, and there are currently no drugs designed for people with an RPE65 mutation that are approved by the FDA.

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However, Luxturna is not without its drawbacks. It’s not an outright cure, and it doesn’t give recipients full 20/20 vision. There’s currently no data on how long its effects last, so there’s a chance that patients’ sight might begin to recede once again over time.

Cost is also a major factor in how accessible it is. Two of the treatment’s biggest competitors, Strimvelis and Kymriah, cost around $700,000 and $475,000 respectively. Consequently it seems likely that Luxturna have to drop in price to be a feasible competitor. They might be losing ground, as Spark has announced plans to set up a program to help patients cover out-of-pocket costs like travel to Spark-proffering facilities.

Gene Therapy

Gene therapy has the potential to make huge improvements to the quality of life of people suffering from various genetic diseases. This blossoming form of treatment could well be the wave of the future.

“This is what I believe medicine is going to be like for the next 20, 30, if not 50 years,” said Spark CEO Jeff Marrazzo, speaking to the MIT Technology Review. “I think this is the beginning of an age that is going to fundamentally change medicine.”

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We’ve already seen projects that use gene therapy to tackle everything from brain diseases to broken bones. However, these treatments will all require FDA approval — so scientists working in this sphere will likely be watching today’s decision regarding Luxturna very closely.


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