Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology

Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform

Ultragenyx to receive $200 million upfront, including $125 million in cash and $75 million via equity investment

NOVATO, Calif., March 31, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo Company, Limited for Ultragenyx’s proprietary AAV-based gene therapy manufacturing technologies. Ultragenyx’s HeLa producer cell line (PCL) platform enables large commercial-scale AAV-based gene therapy product manufacturing that is intended to be highly reproducible, more consistent, and less expensive than other gene therapy manufacturing platforms. In addition, Ultragenyx has developed a proprietary HEK293 transient transfection system for AAV manufacture which is also a subject of the collaboration.

Under the license and technology access agreement, Ultragenyx granted Daiichi Sankyo a non-exclusive license to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. The parties will collaborate closely as part of a technology transfer plan to enable Daiichi Sankyo to use the technologies for its internal gene therapy programs. Ultragenyx retains the exclusive right to use its manufacturing technology for its current target indications and additional indications identified now and in the future.

“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, PhD, Chief Executive Officer 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.”