We don’t follow footsteps.
We create the path.

News & events

FDA advisory committee update
FDA advisory committee recommends approval of investigational LUXTURNA™ (voretigene neparvovec)

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Follow us on Twitter
We’re back on Twitter! Follow us for company updates

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Study published in Clinical and Experimental Ophthalmology
Spark Therapeutics announces publication in Clinical and Experimental Ophthalmology of study evaluating novel test to assess functional vision 

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MAA submitted to the European Medicines Agency
Spark Therapeutics submitted a Marketing Authorization Application for its investigational gene therapy to the European Medicines Agency 

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We don’t follow footsteps. We create the path.

Our mission at Spark Therapeutics is to challenge the inevitability of genetic disease by discovering, developing and delivering potential treatments in ways unimaginable—until now. We have built a fully integrated company, combining our proprietary adeno-associated viral (AAV) gene therapy platform with excellence in R&D, manufacturing and commercial operations. Find out more about how we strive to turn genes into medicines for patients with inherited diseases, including inherited retinal diseases (IRDs), liver-mediated diseases such as hemophilia, and neurodegenerative diseases.

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