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A new biotechnology company will take over human trials of two gene therapies that could offer one-time treatments for a form of childhood blindness and hemophilia B.

The gene therapies were developed by researchers at the Children’s Hospital of Philadelphia, which has committed $50 million to the new company called Spark Therapeutics. The launch is the latest hint that after decades of research and some early setbacks, gene therapy may be on its way to realizing its potential as a powerful treatment for inherited disease.

In December 2012, the European Union gave permission to Dutch company Uniqure to sell its gene therapy for a fat-processing disorder, making Glybera the first gene therapy to make its way into a Western market (see “Gene Therapy on the Mend as Treatment Gets Western Approval”). However, Glybera has not been approved by the U.S., nor has any other gene therapy.

Spark has a chance to be the first gene-therapy company to see FDA approval. Results for a late-stage trial of a gene therapy for Leber’s Congenital Amaurosis, an inherited condition that leads to a loss of vision and eventually blindness, are expected by mid-2015. That treatment is one of several gene therapies in or nearing late-stage testing contending to be the first gene therapy approved by the FDA for sale in the U.S. (see “When Will Gene Therapy Come to the U.S.”).

In addition to taking the reins for two-ongoing human trials, Spark will also work on gene therapies for other eye and blood conditions as well as neurodegenerative diseases, says CEO Jeff Marrazzo.  The gene therapy technology developed at the Children’s Hospital has been “speeding down the tracks,” he says, and the company will provide the “vehicle to get these therapies to the people who need them.”

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Tagged: Biomedicine, gene therapy

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