AChildrens Hospital of Philadelphia spinout with the potential to be the first to win U.S. FDA approval of a gene therapy is now funded through the rest of development of its lead treatment for inherited blindness. Spark Therapeutics is in the middle of a Phase 3 clinical trial that will determine whether the gene therapy its developed can successfully restore some vision to people with rare degenerative eye diseases caused by mutations in the RPE65 gene. Theres currently no treatmentavailable for these diseases. To allow Spark to continue that study and prepare for commercialization, the company has just closed an oversubscribed $72.8 million Series B financing. Investors including Sofinnova Ventures, Brookside, Deerfield, Rock Springs Capital, T. Rowe Price, Wellington and CHOP participated. Developed at CHOPs Center for Cellular and Molecular Therapeutics, the therapy uses a neutralized virus as a vehicle to deliver a function gene to targeted cells in the eye. Once there, it enables production of a critical protein thats missing as a result of a mutation and causes vision loss. In earlier studies, the company reported that some children who were nearly blind as the result of a RPE65 mutation were able to recognize faces and walk without … Continue reading
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