Objective. Recent reports warn that the worldwide cell culture capacity is insufficient to fulfill
the increasing demand for human protein drugs. Production in milk of transgenic animals is
an attractive alternative. Kilogram quantities of product per year can be obtained at relatively
low costs, even in small animals such as rabbits. We tested the long-term safety and efficacy
of recombinant human α-glucosidase (rhAGLU) from rabbit milk for the treatment of the
lysosomal storage disorder Pompe disease. The disease occurs with an estimated …