On this season of America’s Got Talent, the world met Christian Guardino. I was one of millions who heard his beautiful singing voice as he belted out his song onstage. As amazing as his voice was, his story was even more breathtaking. Chrisitian was told as a child that he had a condition that would cause blindness. However, due to a groundbreaking gene therapy, he would later start to regain his sight, according to a post by the DNA Science Blog.
It turns out that Christian has inherited blindness that is specifically called RPE65-mediated inherited retinal dystrophy. The gene therapy that would eventually bring Christian back his eyesight, called Luxturna, started its own journey in 2007 with the launch of a clinical trial. Today, it was just approved by the FDA.
The DNA Science Blog interviewed Dr. Katherine High, founder of Spark Therapeutics, which developed Luxturna. In the interview, she answers some common questions about Luxturna and tells us what the future for gene therapy may hold. Here are a few summarized snippets:
Who can get Luxturna?
People who have two autosomal recessive mutations in the gene RPE65. Patients must also be at least three years old.
What is the process for being treated with Luxturna?
For patients undergoing treatment, one eye is treated at a time to make sure each eye adjusts and progresses accordingly. The second eye is treated about six to 18 days later.
How much does it cost?
The cost is still being determined because several groups are being considered in determining the price, such as patients, policy makers and healthcare providers.
Where will gene therapy be in five years?
As gene therapy starts to become approved in the U.S., in the future, it will take less time for clinical developments to be completed. There has historically been a lack of interest in gene therapy from many physicians; however, as gene therapy products are licensed, there will hopefully be an increased interest about gene therapy in the medical community.
