Profile: GTC Biotherapeutics, Inc. (GTCB.O)

GTC Biotherapeutics, Inc. is engaged in the development and production of human therapeutic proteins through transgenic technology that enables animals to produce a recombinant form of a specified human protein in their milk. Using this technology, the Company is developing a portfolio of recombinant blood proteins to treat a range of genetic and acquired blood deficiencies, including hemophilia and other blood coagulation disorders. These blood proteins, also known as plasma proteins, are difficult to produce in other manufacturing systems, and some are only available by extraction from human blood. It has also initiated the development of a portfolio of monoclonal antibodies (MAb’s), for use as potential follow-on biologics targeted at several market products. Its first product ATryn, its recombinant form of human antithrombin, a blood protein with anticoagulation and anti-inflammatory properties. On February 6, 2009, the Company received United States Food and Drug Administration (FDA), approval for ATryn for HD patients undergoing surgery or childbirth in the United States, making ATryn the first transgenically derived therapeutic protein approved by the FDA.

In June 2008, the Company entered into a collaboration agreement with OVATION Pharmaceuticals to develop and market ATryn in the United States. The collaboration agreement includes the commercialization of ATryn in the HD indication and the further development of ATryn in acquired antithrombin deficiency indications (AD). The Company also have a collaboration agreement with LEO Pharma A/S, or LEO, for the commercialization of ATryn in HD in Europe, Canada, and the Middle East.

The Company is developing a recombinant human coagulation factor VIIa (rhFVIIa), a blood protein for the treatment of hemophilia. The Company obtained a license from ProGenetics, LLC, granting exclusive rights in North America, Europe and Japan to commercially develop recombinant human factor IX (rhFIX), a blood coagulation factor for the treatment of type B hemophilia. An existing rhFIX product marketed as BeneFIX by Wyeth. ProGenetics is responsible for the production of rhFIX in the milk of transgenic pigs. The Company developed goats that produce a recombinant form of human alpha-1 antitrypsin (rhAAT), an inhibitor of elastase. Scientists believe that uninhibited elastase activity in the lungs may be the cause of emphysema and several other respiratory disorders, including chronic obstructive pulmonary disease.

The Company is developing a MAb to the CD20 immune system receptor, which is expected to have specificity similar to Rituximab (Rituxan, Mabthera). This antibody is anticipated to have a relatively higher Antibody Dependent Cell-mediated Cytotoxicity (ADCC), than Rituximab. The Company have provided PharmAthene, Inc. a license to its broad patent for the production of Protexia in transgenic goats. Protexia is a recombinant form of butyrylcholinesterase that is being developed by PharmAthene as a pre- and post-exposure therapy for casualties on the battlefield or civilian victims of nerve agent attacks. In February 2009, the Company entered into a licenses and development agreement with JCOM Co. Ltd., a subsidiary of Dong-A Pharmaceuticals, whereby the Company granted to JCOM an option for an exclusive license for Asia and a separate option for a co-exclusive license for the worldwide, under its patent and know-how rights to make, use, sell, offer for sale and import recombinant human insulin products in these territories.

The Company has developed animals that produce an antibody to CD137, also known as 4-1BB receptor, which is present on T-cells of the human immune system as well as some cancer cells. Its CD137 antibody may have therapeutic value primarily through the modulation of the immune system.

The Company competes with Octopharma, Grifols, Baxter International, Pfizer, Inc., CSL Behring, LFB and Kedrion.