Japan based Daiichi Sankyo Company, Limited has announced that it has entered into a strategic partnership with US based Ultragenyx Pharmaceutical Inc. (hereafter Ultragenyx) for the non-exclusive use of Ultragenyx’s proprietary adeno associated virus (AAV) based gene therapy manufacturing technology.

In the research and development of gene therapy drugs, gene transfer methods to target cells are critical, and the method with the highest expectations is the AAV vector, which can be used to transfer genes into various human tissues.

Daiichi Sankyo is currently doing discovery research for gene therapy drugs using AAV vectors, and in order to deliver gene therapy drugs to patients in the future, it is essential to also establish in-house manufacturing technology at an early stage. Ultragenyx has developed its own AAV production system using HeLa and HEK293 cells, which is particularly excellent in terms of stable quality, high production efficiency, and mass production capability.

Under this agreement, Daiichi Sankyo will complete a technology transfer from Ultragenyx, and following this transfer, Daiichi Sankyo will expand its manufacturing capacity with the cooperation of Ultragenyx and with the intention of starting the manufacture of investigational drugs for AAV gene therapy by the mid-2020s.

Daiichi Sankyo will make an upfront payment of USD $125 million to Ultragenyx at the time of this agreement as well as an additional payment of USD $25 million at the time of the successful technology transfer and sales royalties of products manufactured by Ultragenyx’s technology. Daiichi Sankyo will also purchase Ultragenyx common stock worth USD $75 million.