In Brief
An FDA advisory panel just unanimously approved a new gene therapy that can restore sight in patients with a rare genetic disorder. In clinical trials, more than 90 percent of patients saw improvement in eyesight.

Unanimous Vote

After some emotional testimony from doctors and patients, a Food and Drug Administration (FDA) advisory panel has voted unanimously to approve a gene therapy that improves hereditary blindness. The treatment will now progress to a final decision from the FDA and, if approved, will be the first gene therapy legally available in the United States for an inherited disorder. The FDA is under no obligation to follow the advisory board’s recommendation but usually does.

The treatment, which will be marketed as Luxturna, fixes a mutation in the RPE65 gene. It involves a single treatment to each eye, which introduces genetically engineered virus particles carrying a corrected version of the mutated gene. Spark Therapeutics, the treatment’s developer, estimates that 6,000 people around the world could benefit from this treatment. More than 90 percent of the patients treated in the study showed some improvement in eyesight within just a few days of treatment.

Image credit: Spark Therapeutics
Spark Therapeutics could make history with the first legally available gene therapy in the US. Image Credit: Spark Therapeutics

Gene Therapy Breakthrough

This is a huge step forward for the field of gene therapeutics. “[O]n multiple fronts, it’s a first and ushers in a new era of gene therapy,” assistant professor of ophthalmology at the Oregon Health and Science University, Paul Yang, told NPR.

Alone, this treatment could also be applied to other formally incurable genetic eye diseases. “There are a lot of retinal diseases like this, and if you added them together it’s a big thing because they are all incurable,” says lead researcher Albert Maguire in an interview with NPR before the hearing.

Other gene therapy clinical trials are currently being held around the world. According to the Washington Post, diseases ranging from hemophilia to Huntington’s Disease, an inherited condition that causes the progressive breakdown of nerve cells in the brain, are being targeted for gene therapy treatments.

As with any medical treatment, successful gene therapy doesn’t come without risk. Still, this is huge progress toward correcting previously incurable conditions. This treatment and others like it have the potential to transform countless lives in the very near future.