A Food and Drug Administration advisory committee have come up with a new idea of gene therapy. It is an experimental process to eradicate hereditary disorder such as blindness and as of now they have urged approval for it.
If they get approval for the same then this will be the first time in history for gene therapy in United States.
The process involves injecting RPE65 gene which will help to generate healthy protein required for sight. This will not give 100 percent vision, but will surely improve the vision of an inherited disorder patient. It is been manufactured by Philadelphia-based Spark Therapeutics.
Till date FDA has approved Kymriah which also a gene therapy related to extracting immune cells, developing them in labs and once again injecting them to the patients.
In true sense, Luxturna which is also a Spark product, is known as one of the true gene therapy where a functional gene activates a defective one with its immense power.
RPE65 gene may show its positive result, but according to a report around 1000-2000 people are suffering from retinal diseases just because of a mutated RPE65 gene in United States.
The company has not disclosed the price, but according to experts it may vary from $750,000 to $1 million for both eyes.
Leber congenital amaurosis or retinitis pigmentosa is a hereditary disease that can be cured at an early age if found in children. In this case, children have limited vision which gets worse with due course of time resulting to night blindness. But bad news is that most of them end up with complete blindness.
Recently during the committee meeting, many shared their experiences by saying how the experimental treatment changed their lives. They said that it was amazing to see the stars and their parent’s face for the first time.