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The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world.

Pierce GF, Coffin D; Members of the WFH Gene Therapy Round Table Program Committee and Organizing Committee.

Haemophilia. 2019 Mar;25(2):189-194. doi: 10.1111/hae.13673. Epub 2019 Jan 3. Review.


Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes.

Pierce GF, Iorio A.

Haemophilia. 2018 May;24 Suppl 6:60-67. doi: 10.1111/hae.13489. Review.


Genotypes, phenotypes and whole genome sequence: Approaches from the My Life Our Future haemophilia project.

Konkle BA, Johnsen JM, Wheeler M, Watson C, Skinner M, Pierce GF; My Life Our Future programme.

Haemophilia. 2018 May;24 Suppl 6:87-94. doi: 10.1111/hae.13506. Review.


Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project.

Iorio A, Skinner MW, Clearfield E, Messner D, Pierce GF, Witkop M, Tunis S; coreHEM panel.

Haemophilia. 2018 Jul;24(4):e167-e172. doi: 10.1111/hae.13504. Epub 2018 May 20.


First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries.

Pierce GF, Haffar A, Ampartzidis G, Peyvandi F, Diop S, El-Ekiaby M, van den Berg HM.

Haemophilia. 2018 Mar;24(2):229-235. doi: 10.1111/hae.13409. Epub 2018 Mar 14.


World bleeding disorders registry: The pilot study.

Coffin D, Herr C, O'Hara J, Diop S, Hollingsworth R, Srivastava A, Lillicrap D, van den Berg HM, Iorio A, Pierce GF.

Haemophilia. 2018 May;24(3):e113-e116. doi: 10.1111/hae.13431. Epub 2018 Feb 1. No abstract available.


Novel approach to genetic analysis and results in 3000 hemophilia patients enrolled in the My Life, Our Future initiative.

Johnsen JM, Fletcher SN, Huston H, Roberge S, Martin BK, Kircher M, Josephson NC, Shendure J, Ruuska S, Koerper MA, Morales J, Pierce GF, Aschman DJ, Konkle BA.

Blood Adv. 2017 May 18;1(13):824-834. doi: 10.1182/bloodadvances.2016002923. eCollection 2017 May 23.


AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, Yu H, Vettermann C, Pierce GF, Wong WY, Pasi KJ.

N Engl J Med. 2017 Dec 28;377(26):2519-2530. doi: 10.1056/NEJMoa1708483. Epub 2017 Dec 9.


A Cornucopia of Therapies under Study for Hemophilia.

Pierce GF, Pipe SW.

Mol Ther. 2017 Nov 1;25(11):2429-2430. doi: 10.1016/j.ymthe.2017.10.009. Epub 2017 Oct 19. No abstract available.


Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies.

Pierce GF, Ragni MV, van den Berg HM, Weill A, O'Mahony B, Skinner MW, Pipe SW.

Haemophilia. 2017 Sep;23(5):643-644. doi: 10.1111/hae.13313. Epub 2017 Aug 17. No abstract available.


Evaluation of recombinant factor VIII Fc (Eloctate) activity by thromboelastometry in a multicenter phase 3 clinical trial and correlation with bleeding phenotype.

Driessler F, Miguelino MG, Pierce GF, Peters RT, Sommer JM.

Blood Coagul Fibrinolysis. 2017 Oct;28(7):540-550. doi: 10.1097/MBC.0000000000000638.


Treatment of bleeding episodes with recombinant factor VIII Fc fusion protein in A-LONG study subjects with severe haemophilia A.

Shapiro AD, Mahlangu JN, Perry D, Pasi J, Quon DV, Chowdary P, Tsao E, Li S, Innes A, Pierce GF, Allen GA.

Haemophilia. 2017 May;23(3):392-399. doi: 10.1111/hae.13144. Epub 2017 Feb 20.


Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study.

Fischer K, Kulkarni R, Nolan B, Mahlangu J, Rangarajan S, Gambino G, Diao L, Ramirez-Santiago A, Pierce GF, Allen G.

Lancet Haematol. 2017 Feb;4(2):e75-e82. doi: 10.1016/S2352-3026(16)30193-4.


Long-term safety and efficacy of extended-interval prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) in subjects with haemophilia B.

Pasi KJ, Fischer K, Ragni M, Nolan B, Perry DJ, Kulkarni R, Ozelo M, Mahlangu J, Shapiro AD, Baker RI, Bennett CM, Barnes C, Oldenburg J, Matsushita T, Yuan H, Ramirez-Santiago A, Pierce GF, Allen G, Mei B.

Thromb Haemost. 2017 Feb 28;117(3):508-518. doi: 10.1160/TH16-05-0398. Epub 2016 Dec 22.


Changes in health-related quality of life with treatment of longer-acting clotting factors: results in the A-LONG and B-LONG clinical studies.

Wyrwich KW, Krishnan S, Auguste P, Poon JL, von Maltzahn R, Yu R, Pierce GF, Mei B, Mahlangu J, von Mackensen S.

Haemophilia. 2016 Nov;22(6):866-872. doi: 10.1111/hae.12987. Epub 2016 Jul 7.


Long-acting recombinant factor VIII Fc fusion protein (rFVIIIFc) for perioperative haemostatic management in severe haemophilia A.

Mahlangu JN, Ragni M, Gupta N, Rangarajan S, Klamroth R, Oldenburg J, Nogami K, Young G, Cristiano LM, Dong Y, Allen G, Pierce GF, Robinson B.

Thromb Haemost. 2016 Jul 4;116(1):1-8. doi: 10.1160/TH15-10-0780. Epub 2016 Mar 10.


Recombinant Factor IX Fc Fusion Protein Maintains Full Procoagulant Properties and Exhibits Prolonged Efficacy in Hemophilia B Mice.

Toby GG, Liu T, Buyue Y, Zhang X, Bitonti AJ, Pierce GF, Sommer JM, Jiang H, Peters RT.

PLoS One. 2016 Feb 3;11(2):e0148255. doi: 10.1371/journal.pone.0148255. eCollection 2016.


Recombinant factor VIII Fc (rFVIIIFc) fusion protein reduces immunogenicity and induces tolerance in hemophilia A mice.

Krishnamoorthy S, Liu T, Drager D, Patarroyo-White S, Chhabra ES, Peters R, Josephson N, Lillicrap D, Blumberg RS, Pierce GF, Jiang H.

Cell Immunol. 2016 Mar;301:30-9. doi: 10.1016/j.cellimm.2015.12.008. Epub 2015 Dec 29.


Evaluation of the toxicology and pharmacokinetics of recombinant factor VIII Fc fusion protein in animals.

Dumont JA, Loveday KS, Light DR, Pierce GF, Jiang H.

Thromb Res. 2015 Dec;136(6):1266-72. doi: 10.1016/j.thromres.2015.07.019. Epub 2015 Jul 23.


Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A.

Nolan B, Mahlangu J, Perry D, Young G, Liesner R, Konkle B, Rangarajan S, Brown S, Hanabusa H, Pasi KJ, Pabinger I, Jackson S, Cristiano LM, Li X, Pierce GF, Allen G.

Haemophilia. 2016 Jan;22(1):72-80. doi: 10.1111/hae.12766. Epub 2015 Jul 27.


Manufacturing process used to produce long-acting recombinant factor VIII Fc fusion protein.

McCue J, Kshirsagar R, Selvitelli K, Lu Q, Zhang M, Mei B, Peters R, Pierce GF, Dumont J, Raso S, Reichert H.

Biologicals. 2015 Jul;43(4):213-9. doi: 10.1016/j.biologicals.2015.05.012. Epub 2015 Jun 17.


Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A.

Young G, Mahlangu J, Kulkarni R, Nolan B, Liesner R, Pasi J, Barnes C, Neelakantan S, Gambino G, Cristiano LM, Pierce GF, Allen G.

J Thromb Haemost. 2015 Jun;13(6):967-77. doi: 10.1111/jth.12911. Epub 2015 Apr 23.


Evaluation of the toxicology, pharmacokinetics, and local tolerance of recombinant factor IX Fc fusion protein in animals.

Dumont JA, Loveday KS, Light DR, Pierce GF, Jiang H.

Thromb Res. 2015 Aug;136(2):371-8. doi: 10.1016/j.thromres.2015.01.020. Epub 2015 Jan 23.


Enhanced Pharmacokinetics of Factor VIIa as a Monomeric Fc Fusion.

Salas J, Liu T, Lu Q, Kulman JD, Ashworth T, Kistanova E, Moore N, Pierce GF, Jiang H, Peters R.

Thromb Res. 2015 May;135(5):970-6. doi: 10.1016/j.thromres.2014.12.018. Epub 2015 Jan 3.


A single chain variant of factor VIII Fc fusion protein retains normal in vivo efficacy but exhibits altered in vitro activity.

Buyue Y, Liu T, Kulman JD, Toby GG, Kamphaus GD, Patarroyo-White S, Lu Q, Reidy TJ, Mei B, Jiang H, Pierce GF, Sommer JM, Peters RT.

PLoS One. 2014 Nov 21;9(11):e113600. doi: 10.1371/journal.pone.0113600. eCollection 2014.


Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates.

Powell J, Shapiro A, Ragni M, Negrier C, Windyga J, Ozelo M, Pasi J, Baker R, Potts J, Li S, Mei B, Pierce GF, Robinson B.

Br J Haematol. 2015 Jan;168(1):113-23. doi: 10.1111/bjh.13109. Epub 2014 Sep 11.


Long-acting recombinant factor IX Fc fusion protein (rFIXFc) for perioperative management of subjects with haemophilia B in the phase 3 B-LONG study.

Powell JS, Apte S, Chambost H, Hermans C, Jackson S, Josephson NC, Mahlangu JN, Ozelo MC, Peerlinck K, Pasi J, Perry D, Ragni MV, Wang X, Jiang H, Li S, Cristiano LM, Innes A, Nugent K, Brennan A, Luk A, Allen G, Pierce GF, Robinson B.

Br J Haematol. 2015 Jan;168(1):124-34. doi: 10.1111/bjh.13112. Epub 2014 Sep 11.


Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels.

Shapiro AD, Ragni MV, Kulkarni R, Oldenberg J, Srivastava A, Quon DV, Pasi KJ, Hanabusa H, Pabinger I, Mahlangu J, Fogarty P, Lillicrap D, Kulke S, Potts J, Neelakantan S, Nestorov I, Li S, Dumont JA, Jiang H, Brennan A, Pierce GF.

J Thromb Haemost. 2014 Nov;12(11):1788-800. doi: 10.1111/jth.12723. Epub 2014 Oct 10.


Comparative field study: impact of laboratory assay variability on the assessment of recombinant factor IX Fc fusion protein (rFIXFc) activity.

Sommer JM, Buyue Y, Bardan S, Peters RT, Jiang H, Kamphaus GD, Gray E, Pierce GF.

Thromb Haemost. 2014 Nov;112(5):932-40. doi: 10.1160/TH13-11-0971. Epub 2014 Aug 21.


Validation of the manufacturing process used to produce long-acting recombinant factor IX Fc fusion protein.

McCue J, Osborne D, Dumont J, Peters R, Mei B, Pierce GF, Kobayashi K, Euwart D.

Haemophilia. 2014 Jul;20(4):e327-35. doi: 10.1111/hae.12451. Epub 2014 May 8.


Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B.

Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators.

N Engl J Med. 2013 Dec 12;369(24):2313-23. doi: 10.1056/NEJMoa1305074. Epub 2013 Dec 4.


Comparative field study evaluating the activity of recombinant factor VIII Fc fusion protein in plasma samples at clinical haemostasis laboratories.

Sommer JM, Moore N, McGuffie-Valentine B, Bardan S, Buyue Y, Kamphaus GD, Konkle BA, Pierce GF.

Haemophilia. 2014 Mar;20(2):294-300. doi: 10.1111/hae.12296. Epub 2013 Nov 22.


Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A.

Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators.

Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13.


Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics.

Rath T, Baker K, Dumont JA, Peters RT, Jiang H, Qiao SW, Lencer WI, Pierce GF, Blumberg RS.

Crit Rev Biotechnol. 2015 Jun;35(2):235-54. doi: 10.3109/07388551.2013.834293. Epub 2013 Oct 24. Review.


Endogenous factor VIII synthesis from the intron 22-inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A.

Pandey GS, Yanover C, Miller-Jenkins LM, Garfield S, Cole SA, Curran JE, Moses EK, Rydz N, Simhadri V, Kimchi-Sarfaty C, Lillicrap D, Viel KR, Przytycka TM, Pierce GF, Howard TE, Sauna ZE; PATH (Personalized Alternative Therapies for Hemophilia) Study Investigators.

Nat Med. 2013 Oct;19(10):1318-24. doi: 10.1038/nm.3270. Epub 2013 Sep 15.


Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein.

Peters RT, Toby G, Lu Q, Liu T, Kulman JD, Low SC, Bitonti AJ, Pierce GF.

J Thromb Haemost. 2013 Jan;11(1):132-41. doi: 10.1111/jth.12076.


Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs.

Dumont JA, Liu T, Low SC, Zhang X, Kamphaus G, Sakorafas P, Fraley C, Drager D, Reidy T, McCue J, Franck HW, Merricks EP, Nichols TC, Bitonti AJ, Pierce GF, Jiang H.

Blood. 2012 Mar 29;119(13):3024-30. doi: 10.1182/blood-2011-08-367813. Epub 2012 Jan 13.


Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients.

Powell JS, Josephson NC, Quon D, Ragni MV, Cheng G, Li E, Jiang H, Li L, Dumont JA, Goyal J, Zhang X, Sommer J, McCue J, Barbetti M, Luk A, Pierce GF.

Blood. 2012 Mar 29;119(13):3031-7. doi: 10.1182/blood-2011-09-382846. Epub 2012 Jan 5.


Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients.

Shapiro AD, Ragni MV, Valentino LA, Key NS, Josephson NC, Powell JS, Cheng G, Thompson AR, Goyal J, Tubridy KL, Peters RT, Dumont JA, Euwart D, Li L, Hallén B, Gozzi P, Bitonti AJ, Jiang H, Luk A, Pierce GF.

Blood. 2012 Jan 19;119(3):666-72. doi: 10.1182/blood-2011-07-367003. Epub 2011 Nov 22.


Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Arruda VR, Stedman HH, Haurigot V, Buchlis G, Baila S, Favaro P, Chen Y, Franck HG, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, Nichols TC, High KA.

Blood. 2010 Jun 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24.


Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment.

Mei B, Pan C, Jiang H, Tjandra H, Strauss J, Chen Y, Liu T, Zhang X, Severs J, Newgren J, Chen J, Gu JM, Subramanyam B, Fournel MA, Pierce GF, Murphy JE.

Blood. 2010 Jul 15;116(2):270-9. doi: 10.1182/blood-2009-11-254755. Epub 2010 Mar 1.


Treatment of nonhealing diabetic foot ulcers with a platelet-derived growth factor gene-activated matrix (GAM501): results of a phase 1/2 trial.

Mulder G, Tallis AJ, Marshall VT, Mozingo D, Phillips L, Pierce GF, Chandler LA, Sosnowski BK.

Wound Repair Regen. 2009 Nov-Dec;17(6):772-9. doi: 10.1111/j.1524-475X.2009.00541.x. Epub 2009 Oct 12.


Enhanced efficacy of recombinant FVIII in noncovalent complex with PEGylated liposome in hemophilia A mice.

Pan J, Liu T, Kim JY, Zhu D, Patel C, Cui ZH, Zhang X, Newgren JO, Reames A, Canivel D, Jesmok G, Pierce GF, Sommer JM, Jiang H.

Blood. 2009 Sep 24;114(13):2802-11. doi: 10.1182/blood-2009-03-212423. Epub 2009 Aug 4.


The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene.

Chen L, Zhu F, Li J, Lu H, Jiang H, Sarkar R, Arruda VR, Wang J, Zhao J, Pierce GF, Ding Q, Wang X, Wang H, Pipe SW, Liu XQ, Xiao X, Camire RM, Xiao W.

Mol Ther. 2007 Oct;15(10):1856-62. Epub 2007 Jul 24.


Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.

Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E, Edmonson SA, Hui DJ, Sabatino DE, Zhou S, Wright JF, Jiang H, Pierce GF, Arruda VR, High KA.

Blood. 2007 Oct 1;110(7):2334-41. Epub 2007 Jul 3.


Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment.

Pierce GF, Lillicrap D, Pipe SW, Vandendriessche T.

J Thromb Haemost. 2007 May;5(5):901-6. Review.


CD8(+) T-cell responses to adeno-associated virus capsid in humans.

Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertl HC, High KA.

Nat Med. 2007 Apr;13(4):419-22. Epub 2007 Mar 18.


Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography.

Qu G, Bahr-Davidson J, Prado J, Tai A, Cataniag F, McDonnell J, Zhou J, Hauck B, Luna J, Sommer JM, Smith P, Zhou S, Colosi P, High KA, Pierce GF, Wright JF.

J Virol Methods. 2007 Mar;140(1-2):183-92. Epub 2006 Dec 28.


Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.

Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Mingozzi F, High KA, Pierce GF.

Blood. 2006 Nov 15;108(10):3321-8. Epub 2006 Jul 25.


Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

Jiang H, Pierce GF, Ozelo MC, de Paula EV, Vargas JA, Smith P, Sommer J, Luk A, Manno CS, High KA, Arruda VR.

Mol Ther. 2006 Sep;14(3):452-5. Epub 2006 Jul 5.

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