Rethinking transgenic production
Although production of therapeutic proteins in the milk of transgenic animals holds promise for some complex peptides, it still appears to fall short of the ease of use and scaleability of cell culture production systems. Last week Genzyme General and partner Pharming Group N.V. dropped their transgenically produced alpha-glucosidase enzyme to treat Pompe's disease in favor of a license to a recombinant form of the enzyme produced in CHO cells. The companies determined that the CHO-based system could be scaled up more readily for commercial production than could transgenic rabbits.
GENZ (Cambridge, Mass.) and PHAR (Leiden, the Netherlands) will share development costs and revenues from Pompase equally, following the lines of their 1998 agreement to develop human alpha-glucosidase produced in the milk of transgenic rabbits. As part of the new arrangement, GENZ is lending $10 million to PHAR in exchange for a convertible note that PHAR also can put to its partner...