Spark Therapeutics develops curative, one-time gene therapy products targeting rare, debilitating diseases. Its lead gene therapy candidate for RPE65-related blindness is in Phase 3 clinical trials and could be the first treatment for patients with inherited retinal dystrophies. The company has clinical and preclinical programs in other inherited retinal dystrophies and hematological disorders. Spark also operates a proprietary manufacturing platform that has supported human gene therapy trials across diverse therapeutic areas and routes of administration. It plans to use recent financing to advance its pipeline, including the lead Phase 3 program for RPE65-related retinal dystrophies, and to support company growth over the next three years. Jeffrey D. Marrazzo is co-founder and CEO, and The Children’s Hospital of Philadelphia is a co-founder.

Total raised

$73M

Funding rounds

1

Latest round

Series B

Latest activity

May 2014