When you think of viruses, the yearly flu or even the Ebola or Swine flu outbreaks may come to mind. However, not all viruses cause disease – some even provide cures!

Adeno-associated virus (AAV) can infect humans, but is not known to cause disease. In other words, this virus is good at getting its genetic information (genes) into human cells. What if its genes were replaced with human genes related to disease? Could we cure genetic disease by giving cells a healthy copy of the mutated gene? These questions helped spark the field of gene therapy.

On December 19, 2017, the FDA approved the first directly administered gene therapy in the U.S. to target an inherited genetic disease. This drug, Luxturna, is for patients with a rare form of inherited vision loss that could cause blindness. In this disease, an essential enzyme for normal eye development is missing due to a mutation in the RPE65 gene. Luxturna uses a modified AAV virus to deliver a functional RPE65 gene directly into the retina of the eye. This one-time injection can restore vision long-term.

“Today’s approval marks another first in the field of gene therapy and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases,” said FDA Commissioner Scott Gottlieb, M.D.

The next challenge will be making this drug affordable, as it is very expensive to prepare. Spark Therapeutics, the makers of Luxturna, announced the estimated $825,000 cost earlier this week. The CEO of Spark Therapeutics Jeffrey Marrazzo said “we have been working with health insurers to create innovative pathways for access to Luxturna that may serve as models for other one-time administered gene therapies in the future.”


Managing Correspondent: Chelsea Weidman

Press Articles: First gene therapy for inherited disease gets FDA approval. NPR

Luxturna gene therapy for blindness to cost $825,000. NBC News

Original News Article: FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. U.S. Food & Drug Administration.

Image Credit: Virusworld

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