GTC Biotherapeutics (fka GTCB)

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Risks per GTCB (partial)

http://yahoo.brand.edgar-online.com/fetchFilingFrameset.aspx?dcn=0001193125-06-054015&Type=HTML

"Developing products based on transgenic technology is subject to significant development risks. Each DNA construct is unique and it is possible that it might not be expressed in the transgenic animal’s milk at a level that is commercially viable. Purifying the recombinant protein out of the milk to use as a biotherapeutic may be too difficult to be commercially feasible. In addition, production of the recombinant protein may have negative effects on the health of either the mammary gland or more systematically on the animal as a whole. This would compromise the ability of the animal to produce the recombinant protein. Directing the mammary gland to produce additional proteins in the milk could negatively affect lactation, thereby shutting down milk production. The mammary gland may also modify a protein in such a manner that it is non-functional or harmful to human subjects. It is also possible that there may be disease agents present in goats or cows that would prevent the use of products derived from these animals. If an as yet unknown disease was identified that could not be effectively mitigated, government agencies may confiscate or destroy the animals, or prevent the utilization of their milk. Any of these governmental actions would prevent the use of the recombinant proteins.

To our knowledge, Pharming B.V. is the only other entity to have completed human clinical trials of a transgenically produced product, although Pharming has not filed for marketing authorization in the United States or Europe for any therapeutic protein produced in the milk of transgenic animals. Until we have completed our current pivotal trial and submitted a Biologics License Application, or BLA, to the FDA for ATryn ® , or our MAA for ATryn ® is approved upon successful re-examination in Europe, we will not have confirmation that our ATryn ® trials are sufficient for approval in the United States or Europe. If we are unable to complete all clinical trials that may be required by the FDA or EMEA, or if any of our other transgenically produced proteins in development are not proved to be safe or effective to the satisfaction of regulatory authorities, it would have a material adverse effect on our business and operations. In addition, it is possible that research and discoveries by others could render our transgenic technology obsolete or noncompetitive as a method of production for protein-based therapeutic products."