A novel gene therapy for treating vision loss receives FDA approval

Luxturna (voretigene neparvovec-rzyl) is a novel gene therapy has been approved by US Food and Drug Administration (FDA) this week for treating inherited vision loss in both children and adult patients. Inherited vision loss can ultimately lead to blindness if untreated; however, Luxturna has shown promising results in tackling this condition. US FDA has approved for the first time a directly administered gene therapy. Luxturna targets the mutations of a specific gene, which causes inherited vision loss in children and adults.

The field of gene therapy has received a major boost after receiving approval from US FDAthis week. Now, gene therapy can not only be used to treat cancer but also vision loss; this is an important milestone that has expanded the clinical applications of gene therapy. This year has borne fruit for researchers who have spent decades in developing gene therapy solutions for various illnesses as three gene therapies have been approved by US FDA for the treatment of serious and rare diseases.

In the near future, US FDA officials are hopeful of making gene therapy a conventional mode of treatment as it shows bright prospects in curing serious diseases which cannot tackled by present modalities. In fact, US FDA officials are going to develop a specialized framework for approval novel gene therapies in the years to come. They have promised to come up with new clinical measures that can specifically evaluate and review all aspects of novel gene therapies, which seem to be important breakthroughs in tackling life-threatening diseases.

Many research studies have confirmed that mutation of a biallelic RPE65 gene leads to retinal dystrophy, which further progresses to cause inherited vision loss and ultimately blindness in patients. Officials of US FDA have confirmed that Luxturna is quite effective in treating such patients. Mutations in any one of the 220 different genes can lead to inherited vision loss and blindness. In scientific terms, this condition is known as hereditary retinal dystrophies that cause several retinal disorders due to genetic mutations. Visual dysfunction is a progressive disorder that leads to blindness both in children and adults.

Every year approximately 1000 to 2000 patients in the US are diagnosed with inherited retinal dystrophy, which is caused by mutation of biallelic RPE65 gene. In these patients, both copies of a particular gene (maternal and paternal gene) contain biallelic mutation carriers that cause mutation in the gene. In this case, RPEG5 directs the production of an enzyme that is required for maintaining normal vision. If mutations occur in the gene RPE65, then the activity of RPE65 can be suppressed or stopped completely. Thus, mutations of RPE65 genes can lead to impaired vision and ultimately blindness in patients with retinal dystrophy. Such patients experience progressive loss of vision over a period of time. The sad truth is that loss of vision first hits these patients during puberty or adolescence and ultimately they turn into blind adults.

Luxturna is a novel gene therapy that directly delivers a normal copy of RPE65 gene into retinal cells. A normal protein is then produced by retinal cells with the help of this gene. Due to this protein, light signals are converted into electrical signals in the retina. Thus, patients with inherited vision loss are cured completely as their normal vision gets restored.  Luxturna was prepared from a naturally occurring adenovirus; this virus was modified in the laboratory with the help of DNA techniques. This gene therapy is a vehicle that directly delivers the normal RPE65 gene in human retina and restores the vision of patients with inherited retinal dystrophy. Thus, it prevents many young adolescents and teenagers from turning blind. This has ultimately given hope to thousands of patients who were told that their condition is incurable till date.

Food and Drug Administration (FDA) is an important agency that is affiliated to the U.S Department of Health and Human Services. It is a major regulatory body in public health administration in the USA. Its main function is to assess and evaluate the safety, efficacy, and security of drugs, vaccines, biological products, and medical devices used on humans and animals. Apart from this, US FDA also investigates safety standards of processed foods, cosmetics, tobacco products, and dietary supplements.


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