BioWorld International Correspondent

BRUSSELS, Belgium - The European Medicines Evaluation Agency has agreed to new rules for testing gene transfer medicines. The note for guidance, adopted at the last meeting of the EMEA's top scientific committee, the Committee for Proprietary Medicinal Products quality, covers quality, preclinical and clinical aspects of testing, and comes into force in October.

The new guidance defines gene transfer as "the deliberate introduction of genetic material into somatic cells for therapeutic, prophylactic or diagnostic purposes." It governs the addition and expression of a gene or genes for therapeutic purposes (such as gene transfer products and cancer vaccines), the inoculation of nucleic acids for the purpose of vaccination against foreign antigens (such as DNA vaccination), and the transfer of nucleic acids with the aim of modifying the function or expression of an endogenous gene. And it prohibits the use of primary xenogeneic cells derived from donor animals "until more information on the safety of this approach has been obtained."

It says information on the suitability of the vector system and the delivery system chosen should be provided in support of marketing authorization applications for such products, and for viral vectors, full documentation of the origin, history and biological characteristics of the parental virus should be supplied. Gene transfer medicines should be produced from well-characterized virus seed or cell banks, it urges. Rigorous characterization of the final processed product is essential, and stability should be appropriately established: a minimum of three successive batches of the bulk product should be characterized to determine consistency with regard to purity, identity, potency and safety.

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