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Spark Therapeutics, Inc. focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases. The company is developing SPK-RPE65, which is in Phase III clinical trial for the treatment of inherited retinal dystrophies, a group of rare blinding conditions caused by non-sex linked, or autosomal recessive, mutations in the RPE65 gene. It is also developing SPK-CHM for the treatment of choroideremia; and preclinical programs in development for the treatment of hemophilia A and neurodegenerative diseases, as well as a pipeline of product candidates targeting rare blinding conditions. The company has collaboration agreement with Pfizer, Inc. for the development and commercialization of SPK-FIX product candidates in its gene therapy program for the treatment of hemophilia B; and development and manufacturing collaboration with Genable Technologies Limited for the manufacture and provision of development advice in the development of Genable's therapeutic product candidate, GT038, to treat rhodopsin-linked autosomal dominant retinitis pigmentosa. The company was founded in 2013 and is based in Philadelphia, Pennsylvania.