In return for access to the adeno-associated virus (AAV) platform, Daiichi Sankyo was prepared to offer $200m (€185m) upfront to Ultragenyx.
The platform at the center of the deal is HeLa, a producer cell line platform that allows for the large commercial-scale production of gene therapies. Ultragenyx states that this platform is ‘more consistent’ and ‘less expensive’ in comparison to other gene therapy manufacturing platforms.
Also included in the deal is Ultragenyx’s proprietary HEK293 transient transfection system for AAV manufacture.
Daiichi will receive non-exclusive access to the technology for use in its internal gene therapy projects.
Once technology transfer is completed to Daiichi, the company will then pay an additional $25m. Ultragenyx will receive single-digit royalties on net sales of any Daiichi product manufactured by the technology.
Masayuki Yabuta, head of Daiichi Sankyo’s biologics division, said, “In order to provide these drugs to patients in the future, manufacturing technology must be established early. Ultragenyx's proprietary technology is particularly excellent in terms of stable quality, high production efficiency, and ability to accommodate mass production.”
According to Daiichi, the company is interested in the development of gene therapies for retinal diseases and rare diseases, as well as specifying that it has no interest in gene therapy to insert genetic material into the host genome.
Yabuta added that the company is currently doing discovery research in the gene therapy area, which Daiichi sees as offering long-term business potential once it completes its strategic plan moving toward 2025, marked by a shift towards the development and licensing of oncology-focused assets.
The agreement Ultragenyx has signed with Daiichi features the option for the former company to co-develop and co-commercialize any rare disease product at the investigational new drug stage.