World Federation of Hemophilia Gene Therapy Registry

Barbara A Konkle¹, Donna Coffin², Glenn F Pierce², Cary Clark³, Lindsey George^{4

5}, Alfonso Iorio⁶, Johnny Mahlangu^{3

7}, Mayss Naccache², Brian O'Mahony^{8

9}, Flora Peyvandi^{3

10}, Steve Pipe^{11

12}, Adrian Quartel¹³, Eileen K Sawyer¹⁴, Mark W Skinner¹⁵, Bartholomew Tortella¹⁶, Crystal Watson¹⁷, Ian Winburn¹⁸; Members of the WFH Gene Therapy Registery Steering Committee

Affiliations

¹ Bloodworks NW, Washington Center for Bleeding Disorders, Seattle, WA, USA.
² World Federation of Hemophilia, Montreal, QC, Canada.
³ International Society on Thrombosis and Hemostasis, Carrboro, NC, USA.
⁴ The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
⁵ Univerisity of Pennsylvania, Philadelphia, PA, USA.
⁶ McMaster University, Hamilton, ON, Canada.
⁷ Haemophilia Comprehensive Care Centre, University of the Witwatersrand, NHLS and Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
⁸ Irish Haemophilia Society, Dublin, Ireland.
⁹ European Hemophilia Consortium, Brussels, Belgium.
¹⁰ IRCCS Maggiore Hospital Milan and University of Milan, Milan, Italy.
¹¹ University of Michigan, Pediatrics, Ann Arbor, MI, USA.
¹² National Hemophilia Foundation, New York, NY, USA.
¹³ BioMarin Pharmaceutical Inc, Novato, CA, USA.
¹⁴ uniQure Inc, Lexington, KY, USA.
¹⁵ Institute for Policy Advancement Ltd, Washington, DC, USA.
¹⁶ Spark Therapeutics Inc, Philadelphia, PA, USA.
¹⁷ American Thrombosis and Hemostssasis Network, Chicago, IL, USA.
¹⁸ Pfizer Inc, New York, NY, USA.

Barbara A Konkle et al. Haemophilia. 2020 Jul.

. 2020 Jul;26(4):563-564.

doi: 10.1111/hae.14015. Epub 2020 May 27.

¹ Bloodworks NW, Washington Center for Bleeding Disorders, Seattle, WA, USA.
² World Federation of Hemophilia, Montreal, QC, Canada.
³ International Society on Thrombosis and Hemostasis, Carrboro, NC, USA.
⁴ The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
⁵ Univerisity of Pennsylvania, Philadelphia, PA, USA.
⁶ McMaster University, Hamilton, ON, Canada.
⁷ Haemophilia Comprehensive Care Centre, University of the Witwatersrand, NHLS and Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
⁸ Irish Haemophilia Society, Dublin, Ireland.
⁹ European Hemophilia Consortium, Brussels, Belgium.
¹⁰ IRCCS Maggiore Hospital Milan and University of Milan, Milan, Italy.
¹¹ University of Michigan, Pediatrics, Ann Arbor, MI, USA.
¹² National Hemophilia Foundation, New York, NY, USA.
¹³ BioMarin Pharmaceutical Inc, Novato, CA, USA.
¹⁴ uniQure Inc, Lexington, KY, USA.
¹⁵ Institute for Policy Advancement Ltd, Washington, DC, USA.
¹⁶ Spark Therapeutics Inc, Philadelphia, PA, USA.
¹⁷ American Thrombosis and Hemostssasis Network, Chicago, IL, USA.
¹⁸ Pfizer Inc, New York, NY, USA.

No abstract available

References

1. Peyvandi F, Garagiolal. Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia. Haemophilia. 2019;25:738–746. - PubMed
1. Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. A Molecular Revolution in the Treatment of Hemophilia [published online ahead of print, 2019 Nov 13]. Mol Ther. 2019;S1525-0016(19):30502–30507. - PMC - PubMed
1. Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Prog. 2019;6:1–8. - PMC - PubMed
1. Gitschier J, Wood WI, Goralka TM, et al. Characterization of the human factor VIII gene. Nature. 1984;312:326–330. - PubMed
1. Wood WI, Capon DJ, Simonsen CC, et al. Expression of active human factor VIII from recombinant DNA clones. Nature. 1984;312:330–337. - PubMed