RECOMBINANT PLASMA PROTEINS ATryn® - Recombinant Human Antithrombin Coagulation Factors - Factor VIIa, Factor IX and Factor VIII GTC has entered into a strategic collaboration with LFB Biotechnologies to develop selected recombinant plasma proteins and monoclonal antibodies using GTC's transgenic production platform. The first two programs in this collaboration are for the development of transgenically produced recombinant forms of human factor VIIa and factor IX. Both proteins are clotting factors in the coagulation of blood and are used to treat hemophilia. Factor VIII is also being developed by GTC for the treatment of Type A hemophilia. The preferred treatment strategy is to provide supplemental coagulation factors prophylactically to avoid episodes of excessive bleeding. Factors VIII and IX were recently in-licensed from ProGenetics, LLC for development in North America, Europe, and Japan. Factors VIIa, IX and VIII are all in the development phase at GTC. Currently both plasma-derived and recombinant products are used in treating hemophilia caused by genetic conditions in which patients' failure to express enough coagulation factors on their own may lead to excessive bleeding. The application of transgenic technology may enable the production and pricing of these coagulation factors at appropriate levels for broader utilization, as well as expanding their use to the treatment of the unmet needs of patients in developing countries and to the treatment of other acquired bleeding conditions. Hematology - rhAAT - Recombinant Human Alpha-1 Antitrypsin rhAAT is a recombinant form of the blood protein alpha-1 antitrypsin and is in the development phase at GTC. GTC has established founder transgenic animals that express rhAAT. The plasma form of this product has been used as a treatment for certain forms of emphysema caused by a congenital deficiency of plasma AAT. GTC believes that rhAAT may also be developed as an effective treatment for other diseases, potentially including cystic fibrosis, chronic obstructive pulmonary diseases, acute respiratory syndrome, and severe asthma. Recombinant Human Albumin GTC has formed a joint venture with Fresenius to expand the commercial development opportunities of GTC's Recombinant Human Albumin (rhA) program. The joint venture will manage development of rhA for both the blood expander market and for the use of rhA in the excipient market. Research grade samples are now available for excipient and cell culture use. MONOCLONAL ANTIBODIES Proprietary - CD137 Antibody GTC was awarded an additional grant of $1.4 million in 2006 for its CD137 monoclonal antibody program from the Small Business Innovative Research, or SBIR, program of the National Institutes of Health. This grant will fund process development, characterization of the antibody and efficacy testing in preclinical models in anticipation of future manufacturing for clinical trials. GTC in-licensed the CD137 antibody from the Mayo Clinic and obtained initial SBIR funding in 2004. This product is in the development phase at GTC. The work planned under this additional grant will be performed over two years in collaboration with Dr. Scott Strome at the University of Maryland and Dr. Lieping Chen at the Johns Hopkins School of Medicine. CD137, also known as 4-1BB, is a member of the tumor necrosis factor/nerve growth factor family of receptors and is a surface glycoprotein found on certain cells of the immune system. The agonistic antibody binds to and stimulates CD137 resulting in the strengthening of the otherwise weak immune response to tumors. Follow-On Biologics - CD20 Antibody GTC and LFB Biotechnologies have initiated development of a transgenically produced CD20 monoclonal antibody under an existing agreement between the two companies. The resulting product is expected to have target specificity similar to Rituximab (Rituxan®, Mabthera®) and to have relatively higher antibody dependent cell-mediated cytotoxicity, or ADCC. The transgenically produced CD20 antibody is anticipated to be commercially developed for oncology and auto-immune indications. Rituximab is used in the treatment of B-cell non-Hodgkin's lymphoma, B-cell leukemia and rheumatoid arthritis. It is also under investigation for a range of auto-immune conditions such as systemic lupus erythematosus, immune thrombocytopenic purpura (ITP), and type-1 diabetes.
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