Gene Editing By Spark Therapeutics Shows Promise In Reversing Vision Loss

By Amy Nordrum

Spark Therapeutics announced positive results from a phase three trial to use gene editing to treat a form of inherited vision loss, Oct. 5, 2015. The company launched from the Children’s Hospital of Philadelphia in 2013. Jeffrey M. Vinocur/Wikimedia Commons
Spark Therapeutics is on course to earn the first U.S. approval for a so-called gene therapy. The Philadelphia-based biotech company said Monday that it saw positive results from a late-stage clinical trial to use gene editing to treat a rare form of inherited vision loss that can lead to blindness.
Gene therapy represents a radical new pathway for the potential treatment of hundreds of diseases by replacing malfunctioning genes with normal ones. Researchers and biotech companies have sought to commercialize gene therapy for decades. Spark’s announcement marks the first successful trial for the use of gene therapy to treat genetic diseases.
Spark plans to file an application for approval with the U.S. Food and Drug Administration in 2016. If approved, the technique will be the first therapy to use gene editing for any disease in the U.S. The company is backed by more than $268 million from investors including the Children’s Hospital of Pennsylvania, whose researchers formed the original plans for the company, and money raised in an initial public offering in January.
After the announcement, Spark Therapeutics’ stock jumped 23 percent, or $10.07, to $54 in Monday trading.
Gene editing is a process through which mutated genes are replaced or supplemented with healthy ones that are inserted directly into a patient’s genome. All previous attempts to treat genetic disorders through similar methods have failed to demonstrate improvement or win regulatory approval. Last spring, researchers found that the initial benefits of another form of gene editing that had shown early promise in reversing blindness faltered after about three years……….
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Source: International Business Times