Gene Therapy for Rare Blindness gets FDA Approval
US Food and Drug Administration (FDA) on December 21, 2017, approved a novel gene therapy for treatment of children and adult patients with an inherited vision loss.
The new gene therapy named as Luxturna (voretigene neparvovec-rzyl) is a treatment for retinal dystrophy caused due to specific genetic mutation. “The 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 wide-range of challenging diseases,” said Scott Gottlieb, Commissioner at FDA.
‘Biallelic RPE65’ mutation-associated retinal dystrophy leads to vision loss and may cause blindness in patients. The RPE65 gene regulates function of an enzyme essential for normal vision. Mutations in the RPE65 gene results into low levels of RPE65 activity, which blocks the visual cycle leading to impaired vision. Luxturna delivers a normal copy of the RPE65 gene directly to retinal cells, which can produce the normal protein required to converts the light in to an electrical signal in the retina for restoring vision loss.
According to Advanced Therapy Products Market report published by Coherent Market Insights, gene therapy can aid in treatment of genetically inherited disorders by replacing defective or impaired gene with functional one to restore the normal functions of body. Gene therapy is advised for only those patients who have viable retinal cells as determined by the physicians. The approval of Luxturna to was granted to Spark Therapeutics Inc. “Patients with ‘biallelic RPE65’ mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed,” added Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER).
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