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High Expression Recombinant Factor VIII

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
Program Year/Program:
2010 / STTR
Agency Tracking Number:
R42HL090112
Solicitation Year:
2010
Solicitation Topic Code:
NHLBI
Solicitation Number:
PA09-081
Small Business Information
EXPRESSION THERAPEUTICS
1860 Montreal Road Tucker, GA 30084-
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 2
Fiscal Year: 2010
Title: High Expression Recombinant Factor VIII
Agency: HHS
Contract: 2R42HL090112-02A1
Award Amount: $1,460,129.00
 

Abstract:

DESCRIPTION (provided by applicant): The hemophilias (A and B) are rare bleeding disorders toward which much scientific and medical effort has been devoted. In 1840, blood transfusion was used for the first time to stop post-operative bleeding in a hemophilia patient and in 1968 the first commercial coagulation factor concentrate became available. The cloning of the fVIII gene in 1984 facilitated the development of recombinant fVIII protein products that became commercially available in 1992. This was viewed as a dramatic therapeutic improvement due to the perceived safety advantage recombinant products have over plasma-derived products, which proved responsible for the infection of thousands of patients with hemophilia with human immunodeficiency virus and/or hepatitis C virus during the 1980's. Since the development of recombinant fVIII, progress in the treatment of hemophilia A has slowed. The current limitations to treatment are 1) access to fVIII-replacement products, 2) the cost of fVIII- replacement products, 3) the development of anti-fVIII immune responses that block treatment efficacy and 4) morbidity due to joint disease. Unless the worldwide fVIII supply increases and prices drop significantly, hemophilia A will remain an important heath burden tohuman society. Therefore, the search for improved therapeutics is warranted. One strategy for improving hemophilia A care is to develop improved recombinant- protein products, e.g. manufactured more efficiently or have increased hemostatic efficacy. The mission of Expression Therapeutics is to develop products that will improve the treatment of individuals with hemophilia A. Our technology is based on the identification of sequence elements within fVIII that can be modified to increase its' biosynthesis. The goal of the current study is to provide preclinical data necessary to get FDA approval to conduct a phase 1 human clinical trial using a high-expression fVIII-replacement product that can be manufactured more efficiently than traditional human recombinant fVIII products. These data will support the commercialization of a novel fVIII-replacement product that will improve the treatment of hemophilia A. PUBLIC HEALTH RELEVANCE: Hemophilia A is a bleeding disorder caused by the insufficiency of a bloodclotting factor, termed factor VIII (fVIII). Current treatment for hemophilia relies on infusion of plasma-derived or recombinant fVIII products to restore circulating fVIII activity. Currently, treatment is offered to less than one-third of all patientsdue to excessive product cost. Unless the worldwide fVIII supply increases and prices drop significantly, hemophilia A will remain an important heath burden to human society, and therefore, the search for improved therapeutics is warranted.

Principal Investigator:

Christopher B. Doering
404-727-7988
cdoerin@emory.edu

Business Contact:

Gabriela Denning
678-278-1140
gdenning@expressiontherapeutics.com
Small Business Information at Submission:

EXPRESSION THERAPEUTICS
1860 Montreal Road Tucker, GA -

EIN/Tax ID: 120382893
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
Research Institution Information:
Emory University
1599 Clifton Road, 4th Floor
ATLANTA, GA 30322-
Contact: