(10 Oct 2017) LEADIN:
An advisory panel for the U.S. Food and Drug Administration is set to decide whether to recommend approval of a form of gene therapy which has helped children and adults with hereditary blindness to see.
If the panel advises recommendation on Thursday (12 October 2017) the FDA will have until January 2018 to decide whether to fully approve the therapy.
The therapy is called Luxturna and if approved, it will be the first time the FDA has accepted a gene therapy for an inherited disease.
STORYLINE:
Drug regulators in the US are deciding whether to approve a new gene therapy treatment for an inherited blindness after a clinical trial in which a group of children and adults were successfully treated.
They all had leber congenital amaurosis (LCA), it's caused by a flawed gene and people born with it can't make a protein needed by the retina.
LCA is caused by flaws in a gene called RPE65.
People with LCA lack a protein that helps convert light into signals to the brain that lets us see.
The retina is tissue at the back of the eye that converts light into signals to the brain. This lets us see and understand objects.
People who have the condition see only bright light and blurry shapes and eventually lose all sight.
The treatment, named Luxterna is made by Philadelphia-based Sparks Therapeutics.
The gene therapy works by puncturing a very fine needle into the white part of the eye.
Then a modified virus containing a healthy copy of the gene is injected into the retina.
This allows the cell to make the missing protein.
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