July 17, 2017 6:32 PM By Stephanie Stahl <\/p>\n
PHILADELPHIA (CBS) The FDA will review a breakthrough treatment for inherited diseases thats being developed here in Philadelphia. <\/p>\n
If its approved, this would be the first gene therapy for an inherited disease. <\/p>\n
Mondays announcement is focused on just one treatment but the technology holds tremendous promise. <\/p>\n
Spark Therapeutics in University City is developing the first of its kind gene therapy for a rare inherited eye disease that causes blindness. <\/p>\n
The FDA, in accepting the application, has put the drug Luxturna on a priority status for accelerated review. <\/p>\n
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This is a huge milestone, not just for spark, but for the field of gene therapy, saidJeff Marrazzo of Spark Therapeutics. <\/p>\n
Spark says the therapy works by replacing missing genetic material with a synthetic version. <\/p>\n
Forty-one patients tested the drug, which is a one-time injection. <\/p>\n
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Patients who received Luxturna actually showed a restoration of their functional vision,Marrazzo said. <\/p>\n
The Smedley brothers who live in Bucks County have an inherited form of blindness. While the current Spark drug being reviewed by the FDA wouldnt work for them, it opens a new world of possibility. <\/p>\n
Having two blind kids, initially, that was the most devastating news, Kristin Smedley, their mother said. <\/p>\n
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But Kristen says her devastation has turned into amazement, seeing how well her sons have adjusted. <\/p>\n
Shes excited for the future now that Spark has what appears to be the formula for treating a variety of inherited diseases. <\/p>\n
Seventy percent have no existing treatment, so when you think about potential to apply gene therapy to genetic diseases or inherited diseases, this represents a first step towards many different diseases that could be applied towards this technology,Marrazzo said. <\/p>\n
In addition to the vision treatment, Spark is also working on a therapy for hemophilia. <\/p>\n
The FDA will decide on the blindness drug in January. <\/p>\n
Stephanie Stahl, CBS 3 and The CW Philly 57s Emmy Award-winning health reporter, is featured daily on Eyewitness News. As one of the television industrys most respected medical reporters, Stephanie has been recognized by community and he... <\/p>\n
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FDA Accepts Application For Possible First Gene Therapy for ... - CBS Philly<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" July 17, 2017 6:32 PM By Stephanie Stahl PHILADELPHIA (CBS) The FDA will review a breakthrough treatment for inherited diseases thats being developed here in Philadelphia. If its approved, this would be the first gene therapy for an inherited disease. Continue reading