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Spark Therapeutics is a fully integrated, commercial-stage gene therapy company and a subsidiary of Roche. The company focuses on discovering, developing, and delivering gene therapies for genetic diseases, including blindness, hemophilia, lysosomal storage disorders, and neurodegenerative diseases. Its flagship product, Luxturna, was the first FDA-approved gene therapy for a genetic disease in the United States. Spark utilizes a proprietary adeno-associated virus (AAV) platform to create one-time precision medicines that address the root cause of inherited conditions. Headquartered in Philadelphia, the company was originally spun out of the Children's Hospital of Philadelphia (CHOP) and continues to lead the field in AAV vector engineering and clinical development.
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