Our Scientific Platform and Programs

Spark Therapeutics is developing SPK-8011, an investigational gene therapy for hemophilia A, or factor VIII deficiency. Hemophilia A is a serious and rare inherited hematologic disorder, characterized by mutations in the factor VIII gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. Spark retains global commercialization rights to its SPK-FVIII program for hemophilia A.

Fidanacogene elaparvovec, previously SPK-9001, is an investigational bio-engineered AAV vector utilizing a high-activity factor IX transgene. Hemophilia B is a serious and rare inherited hematologic disorder, characterized by mutations in the factor IX, or FIX gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. Fidanacogene elaparvovec has received both breakthrough therapy and orphan product designations from the U.S. FDA. Spark Therapeutics initiated the ongoing Phase 1/2 clinical trial of fidanacogene elaparvovec that was transitioned to Pfizer in July 2018. At this time, Pfizer announced initiation of a Phase 3 lead-in study. As part of the collaboration, Pfizer assumes sole responsibility for all subsequent pivotal studies, all regulatory activities, manufacturing and potential global commercialization of any products resulting from the hemophilia B gene therapy program.

Spark Therapeutics is developing SPK-GAA, an investigational gene therapy for the potential treatment of Pompe disease. Pompe disease is an oftentimes fatal lysosomal storage disorder and neuromuscular disease, with systemic, multi-organ manifestations resulting from loss of function mutations in the gene encoding acid alpha-glucosidase (GAA). The initial construct for SPK-GAA was in-licensed from Genethon in 2017, and Spark retains global commercialization rights.