About

Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing one-time, life-altering treatments for debilitating genetic diseases. We initially are applying our integrated platform, which was developed over two decades at The Children?s Hospital of Philadelphia (CHOP), to treat rare diseases where no, or only palliative, therapies exist. The gene therapy vectors used in our programs are engineered using an adeno-associated virus, or AAV, which are designed to encapsulate a genetic sequence and have been demonstrated to be effective vehicles for delivering genetic material into targeted cells, providing unique advantages over alternative therapeutic approaches. Our most advanced product candidate, SPK-RPE65, is intended to treat rare blinding conditions caused by mutations in the RPE65 gene, and in earlier clinical studies showed promising evidence of its ability to safely and durably restore vision in patients. In November 2014, we announced that SPK-RPE65 received breakthrough therapy designation from the U.S. Food and Drug Administration. Breakthough 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. We currently are conducting a fully-enrolled, Phase 3 pivotal clinical trial of SPK-RPE65 and we anticipate reporting final results from this trial during the second half of 2015. We are leveraging SPK-RPE65 to address a broad spectrum of other inherited retinal dystrophies, or IRDs, caused by different genetic mutations. Our first such follow-on product candidate is SPK-CHM for the treatment of Choroideremia, for which we have initiated enrollment of a dose-escalating, Phase 1/2 clinical trial. We expect to begin dosing patients in this trial in the first quarter of 2015. We have also established a pipeline of gene therapy candidates to treat hematologic disorders and neurodegenerative disorders, including SPK-FIX, a program for the potential treatment of hemophilia B through a global collaboration with Pfizer Inc., or Pfizer.