- Philadelphia, PA-based Spark Therapeutics (NASDAQ:ONCE) is set for its IPO of 5.5M shares of common stock at $15 - 17.
- The clinical stage gene therapy firm develops treatments for orphan diseases. Its lead product candidate is SPK-RPE65, currently in Phase 3 for the treatment of rare blinding conditions called inherited retinal dystrophies (IRDs) which are caused by mutations in the RPE65 gene. The FDA has designated SPK-RPE65 a Breakthrough Therapy and an Orphan Drug for this indication. The company plans to submit its Biologics License Application (BLA) to the FDA in 2016.
- It has established human proof-of-concept using gene therapy to express a therapeutic gene in the liver as part of its SPK-FIX program for the potential treatment of hemophilia B, which is caused by a mutation in the Factor IX (FIX) gene. Last month, it entered into a global collaboration with Pfizer to develop and commercialize SPK-FIX.