Dirloctogene samoparvovec explained

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]

Notes and References

  1. News: Roche loses spark for gene therapy, axing hemophilia A candidate from pipeline . 8 December 2023.
  2. Mannucci . Pier Mannuccio . Hemophilia treatment innovation: 50 years of progress and more to come . Journal of Thrombosis and Haemostasis . March 2023 . 21 . 3 . 403–412 . 10.1016/j.jtha.2022.12.029. 36858789 . 257276049 . free .
  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 . 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 . 140 . Supplement 1 . 1899–1901 . 10.1182/blood-2022-158903. free .
  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. . 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 . 24 . 20–29 . 10.1016/j.omtm.2021.11.005. free . 8666598 .
  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 . A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds . Blood . 29 November 2018 . 132 . Supplement 1 . 487 . 10.1182/blood-2018-99-115495. 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 . 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 . 140 . Supplement 1 . 10654–10655 . 10.1182/blood-2022-159017. free .

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