GTC Biotherapeutics (fka GTCB)

[shears]

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.

http://digitalcommons.unl.edu/cgi/viewcontent.cgi?filename=0&article=1000&context=chemeng_fundedproposals&type=additional