Spark Therapeutics, Inc. (ONCE) Company Profile
Spark Therapeutics, Inc. focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases. The company develops SPK-RPE65, which is in Phase III clinical trial for the treatment of genetic blinding conditions called inherited retinal diseases caused by non sex-linked, autosomal recessive, or mutations in the RPE65 gene; and SPK-CHM that is in Phase I/II clinical trial for the treatment of choroideremia. It is also developing SPK-FIX program for hemophilia B; SPK-FVIII program to treat hemophilia A; SPK-TPP1 program for the treatment of a form of Batten disease; RhoNova for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa; and SPK-LHON for treating Leber hereditary optic neuropathy, as well as preclinical programs in development for the treatment of Huntington’s disease and other neurodegenerative diseases. The company has collaboration agreement with Pfiz er, Inc. for the development and commercialization of SPK-FIX product candidates in its gene therapy program for the treatment of hemophilia B. Spark Therapeutics, Inc. was founded in 2013 and is headquartered in Philadelphia, Pennsylvania.
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