Company Name: Spark Therapeutics, Inc.
Spark Therapeutics, Inc. is a developer of gene therapies. The company’s initial focus is on rare diseases and its most advanced product candidate is a treatment for rare blinding conditions that are caused by mutations in the RPE65 gene. The company’s pipeline includes product candidates for inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases.
Ticker Symbol: ONCE (NASDAQ)
Company Website: www.sparktx.com
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Headquarters: Philadelphia, PA