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 F8 gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging.

Fidanacogene elaparvovec, previously SPK-9001, is an investigational bio-engineered AAV vector utilizing a high-activity F9 transgene for hemophilia B, or factor IX deficiency. Hemophilia B is a serious and rare inherited hematologic disorder, characterized by mutations in the F9 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. In July 2018, Spark Therapeutics transitioned its ongoing, open-label Phase 1/2 trial of fidanacogene elaparvovec to Pfizer. Pfizer has announced it plans to initiate a Phase 3 lead-in study. As part of the licensing agreement, 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-3006, 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-3006 was in-licensed from Genethon in 2017.