Transgenic animals as drug factories: a new source of recombinant protein therapeutics

The utility of transgenic animal bioreactors for the production of complex therapeutic proteins is based on lower production costs, higher production capacities and safer, pathogen free products. Until gene therapy becomes broadly efficacious, transgenic-derived therapeutics are the most attractive alternative for prophylactic, replacement therapy in genetic disorders, such as haemophilia. Many other disease states need short-term treatment of significant amounts of recombinant proteins that could be made amply available from transgenic animal sources. In addition, transgenic animals will provide an ideal expression system for the production of a portfolio of alternative therapeutics for patient populations developing inhibiting antibodies, for enhanced bioactivity, or for increased plasma clearance times. The FDA approval of a transgenic-derived therapeutic is still pending, but a review of Phase I & II data from antithrombin III from goat milk is encouraging, and companies are continuing to add potential therapeutics to their product pipeline.