Daiichi Sankyo has licensed a commercial-scale gene therapy manufacturing technology from Ultragenyx Pharmaceutical in a $200m deal.

The Japanese firm will use Ultragenyx’s HeLa producer cell line and HEK293 transfection system for adeno-associated virus (AAV) manufacture in its gene therapy development programs.

Masayuki Yabuta, head of Daiichi’s biologics division, set the deal as part of a wider investment in the firm’s gene therapy business.

Image: iStock/vshivkova

“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.”

Co-commercialization

Daiichi will manufacture all products developed using the technologies with Ultragenyx providing strategic consultation on the development of AAV-based gene therapies and other products for rare diseases.

The non-exclusive license also grants Ultragenyx the option to co-develop and co-commercialize rare disease products developed using the technologies at the IND stage.

Under the deal, Daiichi will make an upfront payment of $125 million and will purchase $75 million of Ultragenyx common stock.

The Japanese drug firm will also pay $25 million upon completion of the technology transfer of the HeLa PCL and HEK293 platforms as well as single-digit royalties on net sales of products manufactured in either system.

Tech history

According to an Ultragenyx presentation last year the HeLa producer cell line is scalable up to 2,000L and “enables AAV production for large indications.”

Ultrgenyx added the cell line when it acquired Dimension Therapeutics in 2017, after the termination of latter’s planned merger deal with Regenxbio.

The technology is used in Ultragenyx partnership with Bayer, which is focused on haemophilia A therapy programme.

The HEK293 transient transfection platform operates in the 200-500 `L range and is “Viable for smaller commercial indications” and the production of materials for clinical trials according to Ultragenyx.