Dirloctocogene samoparvovec, also known as SPK-8011, is an experimental gene therapy developed for hemophilia A by Roche and Spark Therapeutics.[1][2][3][4][5][6] It uses an engineered AAV vector to cause liver cells to produce the Factor VIII blood clotting protein.[3]
References
- Roche loses spark for gene therapy, axing hemophilia A candidate from pipeline retrieved 8 December 2023^
- Pier Mannuccio Mannucci. Hemophilia treatment innovation: 50 years of progress and more to come Journal of Thrombosis and Haemostasis, March 2023^
- Stacy E. Croteau, M. Elaine Eyster, Huyen Tran, Margaret V. Ragni, Benjamin J. Samelson-Jones, Lindsey George, Spencer Sullivan, John E.J. Rasko. Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011