[Studies on quality and safety control of drugs for human use from transgenic animals/clone animals]

Recently the pharmaceuticals, which were produced using transgenic animals, have been developed, and will be submitted for registration in nearly future in Japan as well as in USA and EU. In addition, clone animals are also thought to be useful for the productions of the pharmaceuticals. This study has been, therefore, undertaken to establish the technical requirement for registration of the pharmaceuticals. They should be evaluated from the following standpoints: 1) Transgene construct and its characterization; 2) Creation and characterization of the transgenic founder animal; 3) Establishment of a reliable and continuous source of transgenic founder animals; 4) Generation and selection of the production animals; 5) Maintenance of transgenic animals; 6) Recovery and purification of products from transgenic animals; 7) Characterization of products; 8) Process validation/evaluation and in-process test; 9) Specification of products; 10) Stability of products; 11) Preclinical safety evaluation and clinical evaluation. Cloning technology by nuclear transfer of a transformed somatic cell has been already applied to the creation of the transgenic founder animal for the production of pharmaceuticals. The pharmaceuticals produced using the clone animals could be evaluated from almost the same standpoints. However, the flexible evaluation will be also needed depending on the development of the technology.