The Development of Luxturna Brings Hope to Visually Impaired Patients

Jack Hogan, 13, was born with a rare genetic disorder that threatened his vision. Until today, he could not play baseball. Jack’s condition made him completely blind at night. As a result, he had to leave his friends before dark and go home. His parents lived with the knowledge that Jack would one day become blind unless a treatment became available. Luckily, there was a breakthrough in the medical industry, and Jack became the first recipient of the gene therapy that sought to address inherited forms of vision loss. Luxturna, the gene therapy was approved by the FDA last year. It became the first treatment to treat the genetic disorder.

The treatment delivered into Jack’s right eye a synthetic version of the missing gene. After one week, he will have the next surgery on the left eye after which his eyesight is expected to improve. While speaking to Vox, he was excited at not having to go home early. He can sit and watch TV together with his friends. Additionally, he was thrilled with the prospect of playing baseball. Luxturna was developed at the University of Pennsylvania that is currently run by Spark Therapeutics. Well, while the drug comes to address one of the most complex genetic disorders, the price tag attached to it makes it the most expensive drug approved for sale in the U.S. It comes at the price of $425,000 per eye or $850,000 for both eyes.

The drug is an excellent step towards addressing genetic disorders. However, acquiring it remains a problem. On the other hand, the price may not necessarily reflect the benefits to the patient. Statistics show that one in every 4000 Americans is born with an inherited retinal disorder. The disorders can result from several gene mutations. Jack’s disease came as a mutation in RPE65 gene. The gene is vital in the health of the retina. The mutation leads to loss of eyesight as early as infancy. By the age of 20, most are legally blind.

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