Are You a Healthcare Professional?
This European website, initiated and developed by CSL Behring, has two separate sections with the aim to provide information on haemophilia for an international audience, either to European healthcare professionals or to the general public.*
Yes, I am a healthcare professional*
No, I am not a healthcare professional
- Anguela XM, High KA. Entering the modern era of gene therapy. Annu Rev Med. 2019;70:273-288.
- European Medicines Agency. Luxturna. https://www.ema.europa.eu/en/medicines/human/EPAR/luxturna. Accessed November 5, 2021.
- European Medicines Agency. Zolgensma. https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma. Accessed November 5, 2021.
- Food and Drug Administration. Approved Cellular and Gene Therapy Products. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. Accessed September 5, 2023.
- European Medicines Agency (EMA). Roctavian. https://www.ema.europa.eu/en/medicines/human/EPAR/roctavian-0. Accessed January 28, 2023.
- European Medicines Agency (EMA). Hemgenix. https://www.ema.europa.eu/en/medicines/human/EPAR/hemgenix. Accessed January 28, 2023.
- High KA, Roncarolo MG. Gene therapy. N Engl J Med. 2019;381(5):455-464.
- McCain J. The future of gene therapy. Biotechnol Healthc. 2005;2(3):52-54,56-60.
- Friedmann T, Roblin R. Gene therapy for human genetic disease? Science. 1972;175(4025):949-955.
- Muul LM, Tuschong LM, Soenen SL, et al. Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. Blood. 2003;101(7):2563-2569.
- Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. [press release]. Silver Spring, Maryland, USA: US Food and Drug Administration (FDA). https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics. Published January 15, 2019. Accessed November 5, 2021.
- Ghosh S, Brown AM, Jenkins C, Campbell K. Viral vector systems for gene therapy: a comprehensive literature review of progress and biosafety challenges. Appl Biosaf. 2020;25(1):7-18.
- Food and Drug Administration (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee Meeting #70. Toxicity Risks of Adeno-associated Virus (AAV) Vectors for Gene Therapy (GT). Published September 2021.
- Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1-8.
- American Society of Gene and Cell Therapy (ASGCT) and Pharma Intelligence. Q3 2021 Quarterly Data Report on Gene, Cell, & RNA Therapy Landscape. Accessed December 1, 2021.
- European Medicines Agency. Imlygic. https://www.ema.europa.eu/en/medicines/human/EPAR/imlygic. Accessed November 5, 2021.
- European Medicines Agency. Strimvelis. https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis. Accessed November 5, 2021.
- European Medicines Agency. Kymriah. https://www.ema.europa.eu/en/medicines/human/EPAR/kymriah. Accessed November 5, 2021.
- European Medicines Agency. Yescarta. https://www.ema.europa.eu/en/medicines/human/EPAR/yescarta. Accessed November 5, 2021.
- European Medicines Agency. Zynteglo. https://www.ema.europa.eu/en/medicines/human/EPAR/zynteglo. Accessed November 5, 2021.
- European Medicines Agency. Tecartus. https://www.ema.europa.eu/en/medicines/human/EPAR/tecartus. Accessed November 5, 2021.
- European Medicines Agency. Libmeldy. https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy. Accessed November 5, 2021.
- European Medicines Agency. Abecma. https://www.ema.europa.eu/en/medicines/human/EPAR/abecma. Accessed November 5, 2021.
- European Medicines Agency. Skysona. https://www.ema.europa.eu/en/medicines/human/EPAR/skysona. Accessed November 5, 2021.
- Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18(5):358-378.
- FDA Approves First Gene Therapy to Treat Adults with Hemophilia B [press release]. Silver Spring, Maryland, USA: US Food and Drug Administration (FDA). https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b. November 22, 2022. Accessed January 5, 2023.
- Bak RO, Gomez-Ospina N, Porteus MH, Gene Editing on center stage. Trends Genet. 2018;34(8):600-611.
- Ledford H, Callaway E. Pioneers of CRISPR gene editing win chemistry Nobel. Nature. 2020;586:346-347.
- Bulcha JT, Wang Y, Ma H, et al. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021;6(1):53.
- Rangarajan S, Walsh L, Lester W, et al. AAV5-Factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017;377(26):2519-2530.
- Thomas KR, Folger KR, Capecchi MR. High frequency targeting of genes to specific sites in the mammalian genome. Cell. 1986;44:419-428.
- Boston Children’s Hospital. After decades of evolution, gene therapy arrives. https://answers.childrenshospital.org/gene-therapy-history/. Published December 22, 2020. Accessed November 17, 2021.
- Arruda V, Stedman H, Jian H, et al. Correction of hemophilia B phenotype by novel method of regional intravenous delivery of AAV vector to skeletal muscle of hemophilia B dogs. Mol Ther. 2005;11:S233.
- FDA approval brings first gene therapy to the United States [press release]. Silver Spring, Maryland, USA: US Food and Drug Administration (FDA). https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states. Published August 30, 2017. Accessed November 5, 2021.