Spark Therapeutics

Spark Therapeutics receives FDA Breakthrough Therapy designation for SPK-RPE65

Friday, November 7, 2014

Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, has announced that its lead product candidate, SPK-RPE65, has received Breakthrough Therapy designation from the FDA. Breakthrough Therapy designation is intended to expedite the development and review of investigational therapies for serious or life-threatening conditions where initial clinical evidence demonstrates a significant improvement over existing therapies. SPK-RPE65 targets a group of blinding conditions known as inherited retinal dystrophies (IRDs) caused by autosomal recessive mutations in the RPE65 gene, and for which there currently is no pharmacologic treatment available.

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Spark Therapeutics, Genable Technologies collaborate

Wednesday, March 26, 2014

Spark Therapeutics of Philadelphia and Genable Technologies, a privately held Dublin, Ireland-based biopharmaceutical company, are collaborating on Genable’s lead therapeutic GT038, used to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP). Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark. Spark will be the exclusive manufacturer of the product and provide development advice and experience to Genable to help in the ongoing development of GT038. Spark will receive milestone payments and royalties on future sales of GT038.

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Spark Therapeutics appoints Elliott Sigal to board of directors

Friday, February 14, 2014

Spark Therapeutics, a late-stage company developing gene-based medicines for a wide range of debilitating diseases, has appointed Elliott Sigal, M.D., Ph.D., former director, executive vice president and chief scientific officer of Bristol-Myers Squibb, to its board of directors. Elliott led Bristol-Myers Squibb during its ascent as a biopharmaceutical leader and built one of the industry’s most productive R&D organizations through his forward-thinking approach to innovation.

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