FDA Accepts Application For Possible First Gene Therapy for Genetic Disease In US

PHILADELPHIA (CBS) — The FDA will review a breakthrough treatment for inherited diseases that’s being developed here in Philadelphia.

If it’s approved, this would be the first gene therapy for an inherited disease.

Monday’s announcement is focused on just one treatment but the technology  holds tremendous promise.

Spark Therapeutics in University City is developing the first of its kind gene therapy for a rare inherited eye disease that causes blindness.

The FDA, in accepting the application, has put the drug Luxturna on a priority status for accelerated review.

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“This is a huge milestone, not just for spark, but for the field of gene therapy,” said Jeff Marrazzo of Spark Therapeutics.

Spark says the therapy works by replacing missing genetic material with a synthetic version.

Forty-one patients tested the drug, which is a one-time injection.

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“Patients who received Luxturna actually showed a restoration of their functional vision,” Marrazzo said.

The Smedley brothers who live in Bucks County have an inherited form of blindness. While the current Spark drug being reviewed by the FDA wouldn’t work for them, it opens a new world of possibility.

“Having two blind kids, initially, that was the most devastating news,” Kristin Smedley, their mother said.

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But Kristen says her devastation has turned into amazement, seeing how well her sons have adjusted.

She’s excited for the future now that Spark has what appears to be the formula for treating a variety of inherited diseases.

“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.

In addition to the vision treatment, Spark is also working on a therapy for hemophilia.

The FDA will decide on the blindness drug in January.


via 215Today.

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