Imagine a person
with Hemophilia going to a Treatment Center and thanks to hemophilia research,
leaving without Hemophilia. This is at the very heart of our company. We truly
believe that the Cure for Hemophilia is close. Amazing research has been done
that brings us closer to a Cure. We are so blessed to be living in an era when
Hemophilia is treatable and one day even curable.
Thousands of years of suffering, with no effective treatment for Hemophilia,
and then in the last few decades research has exploded.
2011 - Hemophilia Research Study at Wake Forest shows promise
Click here see White Paper discussing Wake Forest Hemophilia Study
1965 Cryoprecipitate used to treat Hemophilia.1
1968 First FIX and FVIII Concentrates.1
1982 FIX Gene cloned.1
1982 FVIII Gene cloned.1
1985 First viral inactivated factor concentrates become available.1
1992 First Recombinant product becomes available.1
1992 First Gene Therapy trials in mice.1
1993-1994 FIX First Gene Therapy in dogs.1
1993 FIX First Gene Therapy in humans 2% factor level increase.
1994 FIX Gene Therapy in dogs up to 300% increase for 1-2 months.2
1995-1996 First FIX Gene therapy in mice and dogs.1
1996 FVIII Gene Therapy in dogs well over therapeutic level
for 1-2 weeks.3
1997 First Recombinant FIX product.1
1998 FVIII Gene Therapy in humans 4% factor level increase.1
1999 FIX Gene Therapy in humans B 1.6% increase.1
1999 FVIII Gene Therapy in mice produce 20 % increase for 11
2000 FVIII Gene Therapy in mice 4%-20% increase.5
2001 FIX Gene Therapy in humans 2%- 12% increase.1
2001 FVIII Gene Therapy in humans up to 3% increase.1
2002 FVIII Gene Therapy in mice up to 20 % increase.6
2003 FIX Gene Therapy in neonatal mice 150%-280% for more than
2003 FIX Gene Therapy in neonatal dogs
12%- 36% increase for more than 14 Months.7
2004 FVIII Gene Therapy in mice produces 100% correction.8
2004 Vwb Factor Genetically produced in culture.9
2004 FIX Gene therapy in dogs 4%-14% increase for 3 years,
2005 Glannzman's Thrombasthenia Gene Therapy produces therapeutic
levels in mice.11
2005 FVIII Gene Therapy in newborn mice and dogs produces 115%
- 139% factor levels for 1 1/2 years, observation ongoing.12
2006 Gene Therapy produces FVIII stored in platelets to overcome
2006 FVIIA Gene Therapy in mice produces therapeutic levels
to overcome inhibitors.15
2006 FIX produced from microcapsule implanted in mice.16
2006 FIX Gene Therapy in mice produces Long Term Therapeutic
2007 FIX Gene Therapy in humans produces increases for over
3.7 years observation ongoing.18
2007 FVIII Gene Therapy in dogs and mice produces factor level
increases 2%-4.5% for 27 weeks.19
2007 Vwb Factor produced by Gene Therapy.20
2007 FIX Gene Therapy in mice produces over 10% for more than
2006 FVIIa Gene Therapy in mice produces FVIIa.13
1 C. Hough, D. Lillicrap
(2005) Gene therapy for hemophilia: an imperative to succeed, Journal of Thrombosis
and Haemostasis 3 (6), 1195-1205. doi:10.1111/j.1538-7836.2005.01401.x
2 Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wiehle
S, Fang B, Bellinger D, Finegold M, Thompson AR, et al. In vivo hepatic gene
therapy: complete albeit transient correction of factor IX deficiency in hemophilia
B dogs.1: Proc Natl Acad Sci U S A. 1994 Mar 15;91(6):2353-7.
3 S Connelly, J Mount, A Mauser, JM Gardner, M Kaleko, A McClelland,
and CD Jr Lothrop Complete short-term correction of canine hemophilia A by in
vivo gene therapy Blood, Nov 1996; 88: 3846 - 3853.
4 Lang, H Study points to possible cure for hemophilia UNC-CH
School of Medicine News Services June 9, 1999--No.369
5 Steven S. Fakharzadeh, Yue Zhang, Rita Sarkar, and Haig H.
Kazazian, JrCorrection of the coagulation defect in hemophilia A mice through
factor VIII expression in skin Blood, May 2000; 95: 2799 - 2805.
6 Arnette R. New gene therapy treatment works in mice with
hemophilia A. UNC School of Medicine News Services June 10, 2002 -- No. 332
7 Lingfei Xu, Cuihua Gao, Mark S. Sands, Shi-Rong Cai, Timothy
C. Nichols, Dwight A. Bellinger, Robin A. Raymer, Stephanie McCorquodale, and
Katherine Parker Ponder Neonatal or hepatocyte growth factor-potentiated adult
gene therapy with a retroviral vector results in therapeutic levels of canine
factor IX for hemophilia B, Blood, May 2003; 101: 3924 - 3932.
8 Rita Sarkar, Renee Tetreault, Guangping Gao, Lili Wang, Peter
Bell, Randy Chandler, James M. Wilson, and Haig H. Kazazian, Jr Total correction
of hemophilia A mice with canine FVIII using an AAV 8 serotype Blood, Feb 2004;
103: 1253 -1260.
9 Basu, U. Banerjee, S. An engineered EBV vector expressing
human factor VIII and von Willebrand factor in cultured B-cells. J Gene Med.
10 Valder R. Arruda, Hansell H. Stedman, Timothy C. Nichols,
Mark E. Haskins, Matthew Nicholson, Roland W. Herzog, Linda B. Couto, and Katherine
A. High Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves
long-term correction of hemophilia B in a large animal model Blood, May 2005;
105: 3458 - 3464.
11 Juan Fang, Kairbaan Hodivala-Dilke, Bryon D. Johnson, Lily
M. Du, Richard O. Hynes, Gilbert C. White, II, and David A. Wilcox Therapeutic
expression of the platelet-specific integrin, aIIbB3 in a murine model for Glanzmann
thrombasthenia Blood, Oct 2005; 106: 2671 - 2679.
12 Lingfei Xu, Timothy C. Nichols, Rita Sarkar, Stephanie McCorquodale,
Dwight A. Bellinger, and Katherine P. Ponder Absence of a desmopressin response
after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed
neonatal gene therapy PNAS 2005 102: 6080-6085; published online before print
13 Paris Margaritis, Valder R. Arruda, Majed Aljamali, Rodney
M. Camire, Alexander Schlachterman, and Katherine A. High Novel therapeutic
approach for hemophilia using gene delivery of an engineered secreted activated
Factor VII J. Clin. Invest., Apr 2004; 113: 1025 - 1031
14 Qizhen Shi, David A. Wilcox, Scot A. Fahs, Hartmut Weiler,
Clive W. Wells, Brian C. Cooley, Drashti Desai, Patricia A. Morateck, Jack Gorski,
and Robert R. Montgomery Factor VIII ectopically targeted to platelets is therapeutic
in hemophilia A with high-titer inhibitory antibodies J. Clin. Invest., Jul
2006; 116: 1974 - 1982.
15 Margaritis P, High, KA Advances in gene therapy using factor
VIIa in hemophilia. Seminars in Hematology. 2006 Jan (1Supp 1) : S101-4
16 Jianping Wen , Andrew GÅ\mez Vargas , Frederick A.
Ofosu , Gonzalo Hortelano Sustained and therapeutic levels of human factor IX
in hemophilia B mice implanted with microcapsules: key role of encapsulated
cells, The Journal of Gene Medicine 2006 Mar;8(3):362-9
17 Bigger BW, Siapati EK, Mistry A, Waddington SN, Nivsarkar
MS, Jacobs L, Perrett R, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes
SJ, Coutelle C, Wright N, Alison M, Thrasher AJ, Bonnet D, Themis M. Permanent
partial phenotypic correction and tolerance in a mouse model of hemophilia B
by stem cell gene delivery of human factor IX. Gene Ther. 2006 Jan;13(2):117-26.
18 Jiang H, Pierce GF, Ozelo MC, de Paula EV, Vargas JA, Smith
P, Sommer J, Luk A, Manno CS, High KA, Arruda VR. Evidence of multiyear factor
IX expression by AAV-mediated gene transfer to skeletal muscle in an individual
with severe hemophilia B. Mol Ther. 2006 Sep;14(3):452-5. Epub 2006 Jul 5.
19 Matsui H, Shibata M, Brown B, Labelle A, Hegadorn C, Andrews
C, Hebbel RP, Galipeau J, Hough C, Lillicrap D. Ex Vivo Gene Therapy for Hemophilia
A That Enhances Safe Delivery and Sustained In Vivo FVIII Expression From Lentivirally-engineered
Endothelial Progenitors. Stem Cells. 2007 Jul 5; [Epub ahead of print]
20 Park SW, Choi SY. Long-Term Expression of von Willebrand
Factor by a VSV-G Pseudotyped Lentivirus Enhances the Functional Activity of
Secreted B-Domain-deleted Coagulation Factor VIII. Mol Cells. 2007 Aug 31;24(1):125-31
21 Brian D Brown, Alessio Cantore, Andrea Annoni, Lucia Sergi
Sergi, Angelo Lombardo, Patrizia Della Valle, Armando D'Angelo, and Luigi Naldini
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia
B mice Blood, Aug 2007; doi:10.1182/blood-2007-03-078493
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