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Spark Therapeutics L.L.C., a Philadelphia company developing gene-based medicines for debilitating diseases, has signed a collaborative partnership with a gene-therapy firm in Ireland to develop a product to treat a rare form of blindness, the companies announced Tuesday. <\/p>\n
Spark, a biotechnology company spun out of research at Children's Hospital of Philadelphia, said Dublin-based Genable Technologies Ltd. will license certain patents from Spark, which will be the exclusive manufacturer and provide development expertise for a potential treatment for blindness caused by inherited retinal dystrophy. <\/p>\n
Spark will receive milestone payments and royalties on future sales, as well as revenue from the manufacturing and supply of the product. Financial terms were not disclosed. <\/p>\n
Children's Hospital has committed to investing up to $50 million in Spark, which seeks to be the nation's first commercial provider of gene therapy. <\/p>\n
Genable's therapeutic strategy \"knocks down\" or suppresses the \"over-expression of one gene\" causing a disease, while at the same time \"replacing something else that is missing in the patient,\" Marrazzo said in an interview. <\/p>\n
Spark is developing two potential products to treat inherited retinal disease. One is in a late-stage, or Phase 3, clinical trial, \"which is likely to be the first approved gene therapy in the United States,\" Marrazzo said. <\/p>\n
A second drug candidate for retinal disease will begin clinical trials shortly. \"We haven't announced the specifics of that program yet,\" he said. <\/p>\n
Initial results on its lead drug candidate in late-stage testing have been promising, Spark said: Once-blind patients could recognize faces and were moved out of Braille classrooms. They were no longer considered legally blind, and some were able to drive a car. <\/p>\n
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Biotech company Spark Therapeutics, Irish firm to partner on blindness drug<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" Spark Therapeutics L.L.C., a Philadelphia company developing gene-based medicines for debilitating diseases, has signed a collaborative partnership with a gene-therapy firm in Ireland to develop a product to treat a rare form of blindness, the companies announced Tuesday. Spark, a biotechnology company spun out of research at Children's Hospital of Philadelphia, said Dublin-based Genable Technologies Ltd Continue reading