Spark Therapeutics is developing potentially curative, one-time gene therapy products to transform the lives of patients and re-imagine the treatment of debilitating diseases. The founding team includes scientists who have led the advancement of gene therapy over the past two decades, establishing human proof of concept of the expression of gene therapy in the eye and liver. Spark’s robust pipeline includes SPK-RPE65, a fully enrolled, pivotal Phase 3 program in blindness due to mutations in the RPE65 gene, SPK-CHM for the treatment of choroideremia, and SPK-FIX, a program for the potential treatment of hemophilia B through a global collaboration with Pfizer Inc., as well as preclinical programs to address neurodegenerative diseases and other retinal degenerative diseases and hematologic disorders. Spark has rights to a proprietary manufacturing platform that has a track record supporting clinical studies across diverse therapeutic areas and routes of administration. Spark’s expertise across research, clinical, regulatory and manufacturing builds on a legacy of innovation and excellence in gene therapy established by Spark’s team while at The Children’s Hospital of Philadelphia Center for Cellular and Molecular Therapeutics.
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