Ron Philip, Chief Commercial Officer of Spark Therapeutics, discusses his company's pipeline which is focused on a range of debilitating genetic diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia and lysosomal storage disorders and neurodegenerative diseases. Each of Spark's research programs uses an adeno-associated viral (AAV) vector.

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.

SPK-8011 is 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. Spark retains global commercialization rights to its SPK-FVIII program includes both SPK-8011 and SPK-8016 for hemophilia A.

SPK-3006 is 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, and Spark retains global commercialization rights.

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