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 Blood, 15 November 2022^
- Liron Elkouby, Sean M. Armour, Raffaella Toso, Marti DiPietro, Robert J. Davidson, Giang N. Nguyen, Mallory Willet, Stephanie Kutza. Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A Molecular Therapy - Methods & Clinical Development, March 2022^
- Katherine A. High, Lindsey A. George, M. Elaine Eyster, Spencer K. Sullivan, Margaret V. Ragni, Stacy E. Croteau, Ben J. Samelson-Jones, Matthew Evans. A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds Blood, 29 November 2018^
- Matthew S. Evans, Witold B. Rybka, Stacy E. Croteau, Huyen Tran, John E.J. Rasko, Kristen Jaworski, Amy MacDougall, Savina Jaeger. The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011 Blood, 15 November 2022^