Dirloctogene samoparvovec

Dirloctogene 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.
^ Mannucci, Pier Mannuccio (March 2023). "Hemophilia treatment innovation: 50 years of progress and more to come". Journal of Thrombosis and Haemostasis. 21 (3): 403–412. doi:10.1016/j.jtha.2022.12.029. PMID 36858789. S2CID 257276049.
^ ^a ^b Croteau, Stacy E.; Eyster, M. Elaine; Tran, Huyen; Ragni, Margaret V.; Samelson-Jones, Benjamin J.; George, Lindsey; Sullivan, Spencer; Rasko, John E.J.; Moormeier, Jill; Angchaisuksiri, Pantep; Teitel, Jerome; Kenet, Gili; Wynn, Tung; Jaworski, Kristen; Macdougall, Amy; Jaeger, Savina; Trivedi, Trupti; Mingozzi, Federico; Chang, Tiffany; Levy, Gallia (15 November 2022). "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. 140 (Supplement 1): 1899–1901. doi:10.1182/blood-2022-158903.
^ Elkouby, Liron; Armour, Sean M.; Toso, Raffaella; DiPietro, Marti; Davidson, Robert J.; Nguyen, Giang N.; Willet, Mallory; Kutza, Stephanie; Silverberg, Joseph; Frick, Jennifer; Crosariol, Marco; Wang, Yuhuan; Wang, Chuansong; High, Katherine A.; Sabatino, Denise E.; Anguela, Xavier M. (March 2022). "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. 24: 20–29. doi:10.1016/j.omtm.2021.11.005. PMC 8666598. PMID 34977269.
^ High, Katherine A.; George, Lindsey A.; Eyster, M. Elaine; Sullivan, Spencer K.; Ragni, Margaret V.; Croteau, Stacy E.; Samelson-Jones, Ben J.; Evans, Matthew; Joseney-Antoine, Marcelyne; Macdougall, Amy; Kadosh, Judith; Runoski, Alexa R.; Campbell-Baird, Cynthia; Douglas, Kayla; Tompkins, Summer; Hait, Howard; Couto, Linda B.; Bassiri, Ashlyn Eaton; Valentino, Leonard A.; Carr, Marcus E.; Hui, Daniel J; Wachtel, Katie; Takefman, Daniel; Mingozzi, Federico; Anguela, Xavier M.; Reape, Kathleen B (29 November 2018). "A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds". Blood. 132 (Supplement 1): 487. doi:10.1182/blood-2018-99-115495. S2CID 81439244.
^ Evans, Matthew S.; Rybka, Witold B.; Croteau, Stacy E.; Tran, Huyen; Rasko, John E.J.; Jaworski, Kristen; MacDougall, Amy; Jaeger, Savina; Mingozzi, Federico; Chang, Tiffany; Levy, Gallia (15 November 2022). "The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011". Blood. 140 (Supplement 1): 10654–10655. doi:10.1182/blood-2022-159017.

[1] "Roche loses spark for gene therapy, axing hemophilia A candidate from pipeline". Retrieved 8 December 2023.

[2] Mannucci, Pier Mannuccio (March 2023). "Hemophilia treatment innovation: 50 years of progress and more to come". Journal of Thrombosis and Haemostasis. 21 (3): 403–412. doi:10.1016/j.jtha.2022.12.029. PMID 36858789. S2CID 257276049.

[:0-3] Croteau, Stacy E.; Eyster, M. Elaine; Tran, Huyen; Ragni, Margaret V.; Samelson-Jones, Benjamin J.; George, Lindsey; Sullivan, Spencer; Rasko, John E.J.; Moormeier, Jill; Angchaisuksiri, Pantep; Teitel, Jerome; Kenet, Gili; Wynn, Tung; Jaworski, Kristen; Macdougall, Amy; Jaeger, Savina; Trivedi, Trupti; Mingozzi, Federico; Chang, Tiffany; Levy, Gallia (15 November 2022). "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. 140 (Supplement 1): 1899–1901. doi:10.1182/blood-2022-158903.

[4] Elkouby, Liron; Armour, Sean M.; Toso, Raffaella; DiPietro, Marti; Davidson, Robert J.; Nguyen, Giang N.; Willet, Mallory; Kutza, Stephanie; Silverberg, Joseph; Frick, Jennifer; Crosariol, Marco; Wang, Yuhuan; Wang, Chuansong; High, Katherine A.; Sabatino, Denise E.; Anguela, Xavier M. (March 2022). "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. 24: 20–29. doi:10.1016/j.omtm.2021.11.005. PMC 8666598. PMID 34977269.

[5] High, Katherine A.; George, Lindsey A.; Eyster, M. Elaine; Sullivan, Spencer K.; Ragni, Margaret V.; Croteau, Stacy E.; Samelson-Jones, Ben J.; Evans, Matthew; Joseney-Antoine, Marcelyne; Macdougall, Amy; Kadosh, Judith; Runoski, Alexa R.; Campbell-Baird, Cynthia; Douglas, Kayla; Tompkins, Summer; Hait, Howard; Couto, Linda B.; Bassiri, Ashlyn Eaton; Valentino, Leonard A.; Carr, Marcus E.; Hui, Daniel J; Wachtel, Katie; Takefman, Daniel; Mingozzi, Federico; Anguela, Xavier M.; Reape, Kathleen B (29 November 2018). "A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds". Blood. 132 (Supplement 1): 487. doi:10.1182/blood-2018-99-115495. S2CID 81439244.

[6] Evans, Matthew S.; Rybka, Witold B.; Croteau, Stacy E.; Tran, Huyen; Rasko, John E.J.; Jaworski, Kristen; MacDougall, Amy; Jaeger, Savina; Mingozzi, Federico; Chang, Tiffany; Levy, Gallia (15 November 2022). "The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011". Blood. 140 (Supplement 1): 10654–10655. doi:10.1182/blood-2022-159017.

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