Gene Therapy for Vision Loss Approved by FDA

A gene therapy treatment for the disease known as “biallelic RPE65 mutation-associated retinal dystrophy,” a rare condition with onset in childhood or adolescence that leads to complete vision loss, was approved by the FDA in December of 2017. This disease affects approximately 200 Canadians. The treatment, developed by the Pennsylvania-based company Spark Therapeutics Inc., is known as Luxturna (voretigene neparvovecrzyl) and could be used to treat 6000 people worldwide.

“Today’s approval marks another first in the field of gene therapy—both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss—and this milestone reinforces the potential of this breakthrough approach in treating a widerange of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, M.D.

Luxturna functions by delivering a working copy of the RPE65 gene to the retina, which then can produce the normal protein,

allowing the retina to function properly. The treatment is currently extremely expensive, costing $425,000 USD per eye.

The treatment is not yet approved by Health Canada.

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