Chemical and Biomolecular Engineering Departmental Papers

 

Date of this Version

September 2005

Abstract

Three specific aims are proposed: Specific Aim # 1. Process engineer and scale-up the recovery and purification of transgenic recombinant human Factor IX. The University of Nebraska-Lincoln Biological Process Development Facility will complete process development and scale-up, and produce clinical grade materials for preclinical studies. The endpoint is a proposed final product specification to help facilitate transfer to current Good Manufacturing Practices compliant production of clinical grade material to support an Investigational New Drug filing with the United States Food and Drug Administration (FDA) leading to clinical trials. Specific Aim #2. Characterize and formulate transgenic recombinant human Factor IX for intravenous dosage, and evaluate in a hemophilia B dog model. These activities are directed toward characterization of the product important to assure the provision of safe and reproducibly effective hemostasis. The results of these investigations will help support an IND filing with the FDA. Specific Aim # 3. Develop an oral dosage form of transgenic recombinant human Factor IX, and evaluate in hemophilia B mice and dog models. Oral administration of coagulation therapy will obviate the invasiveness, discomfort, potential for opportunistic infection, and complications of storage and supplies that accompany intravenous administration. Oral dosage forms of Factor IX will thus greatly increase the proportion of the patient population that can be treated. There is also published evidence suggesting that oral administration may reduce the potential for complicating immune responses to replacement therapy, especially in patients with severe hemophilia.

NIH grant proposal-Word copy.doc (4652 kB)
NIH grant proposal-Word copy.doc

Share

COinS