First Gene Therapy to Treat an Inherited Disease is Approved

Posted on at February 22, 2018
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Image courtesy of Spark Therapeutics Inc.

The first gene therapy to treat an inherited disease was approved by the Food and Drug Administration on December 19, 2017.

The treatment uses a genetically modified virus (adeno-associated virus) to carry a healthy gene into the eyes of retinal dystrophy patients, thus restoring their ability to see.
Developed by Spark Therapeutics, the therapy—called Luxturna—treats retinal dystrophy, a rare inherited form of blindness.

“This milestone reinforces the potential of this breakthrough approach in treating a wide range of challenging diseases,” said FDA Commissioner Scott Gottlieb in a press release; the treatment does, indeed, have implications for Huntington disease (HD).

A study conducted by Spark Therapeutics involving 31 participants found Luxturna to be effective, with more than 90 percent of the treated patients showing at least some improvement in their vision, often within a few days of treatment. Specifically, Phase 3 of the study examined study participants’ abilities to navigate an obstacle course at various light levels. The group that received Luxturna showed significant improvements in their ability to complete the obstacle course at low light levels compared to the control group.

Spark Therapeutics has followed its patients for more than three years and has found that the treatment’s effects to be long-standing. The team found no significant adverse reactions to the gene therapy.

In a previous interview with HD Insights, Katherine High, president and chief scientific officer at Spark Therapeutics, said that the treatment’s approval by the FDA would signify
“fulfillment of a career spent trying to establish a basis for gene therapy for genetic diseases.” Upon learning of the treatment’s approval by the FDA, she noted that it is time to pursue a gene therapy for HD. In the near future, she said, “I really believe that genes will become medicines.”

For more information about HTTRX, read our interview with Katherine High: huntingtonstudygroup.org/hd-insights/meet-the-company-spark-therapeutics/

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