Daiichi Sankyo Invests in Ultragenyx’s Technology to Bolster Gene Therapy Pipeline
By Rajaneesh K. Gopinath, Ph.D.
Japan’s second-largest drugmaker, Daiichi Sankyo announced its entry into a strategic partnership with California-based Ultragenyx Pharmaceutical Inc., a company that develops innovative technologies and treatments for rare diseases. Daiichi will license Ultragenyx’s adeno associated virus (AAV) based gene therapy manufacturing technology for a total of US$ 225 million. That includes an upfront cash payment of US$ 125 million on signing the agreement and US$ 75 million in equity investment at US$ 60 per share, which is a 35% premium on Ultragenyx’s stock price yesterday. Besides, an additional US$ 25 million will be paid on completion of tech transfer.
Ultragenyx’s Expertise in AAV Technology
Adeno-associated virus (AAV) is the most prevalent mammalian vector used for gene therapies. It has several advantages such as low immunogenicity, lack of pathogenicity in humans, and the ability to stably integrate into the host cell genome and establish long-term gene expression in both dividing and non- dividing cells. Ultragenyx is well known for its AAV products and has its own HeLa and HEK293 cell platform that produces high-quality AAV vectors. Just yesterday, it announced an agreement with long time partner, REGENXBIO Inc. to exclusively license the latter’s novel AAV (NAV) vectors, AAV8 and AAV9 for the development and commercialization of gene therapy treatments for a rare metabolic disorder.
“This new partnership with Daiichi Sankyo provides further validation of the value of Ultragenyx’s gene therapy-related technologies, especially our HeLa producer cell line platform that we believe is the most scalable mammalian cell AAV manufacturing system,” said Emil D. Kakkis, MD, Ph.D., CEO and President of Ultragenyx. “We are encouraged that our proprietary technologies continue to enable the development of numerous programs, both internally and for our partners, to treat patients with serious unmet medical needs.” he added.
Daiichi Ventures into Gene Therapy
Daiichi’s goal is to become a “Global Pharma Innovator with Competitive Advantage in Oncology” but is also setting its sights on the gene therapy market to grow beyond its 2025 vision.
“We are currently doing discovery research for gene therapy drugs using AAV vectors as one of our focused modalities toward sustained growth beyond achievement of our 2025 vision,” said Masayuki Yabuta, Ph.D., Executive Officer, Head of Biologics Division, Daiichi Sankyo. “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.”
As per the agreement, Daiichi is responsible for the manufacture, development, and commercialization of products that come out of using the newly acquired AAV technology, but Ultragenyx will provide strategic consultation and also have the option to co-develop and co-commercialize rare disease products at the IND stage.
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