Ultragenyx Enters Into Two Gene Therapy Deals

Ultragenyx Pharmaceutical signed two rare disease gene therapy deals—one for in-licensing AAV technology and one for out-licensing manufacturing technology.

Photo: Emil Kakkis, president and CEO of Ultragenyx

The first deal is for vector technology, and extends Ultragenyx’ existing gene therapy partnership with Regenxbio. Under the terms of their agreement, Regenxbio granted Ultragenyx an exclusive, worldwide license and rights to sublicense, its AAV8 and AAV9 vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder. In return for these rights, Regenexbio will receive an upfront payment of $7 million, ongoing fees, milestone payments, and royalties on net sales of products incorporating the licensed vectors.

“This latest license agreement reinforces Ultragenyx’s commitment to developing gene therapies for multiple rare diseases and adds a new indication to our relationship with Regenxbio,” said Emil Kakkis, president and CEO of Ultragenyx.

The companies have been long-standing partners, and Ultragenyx has used Regenxbio’s vectors for the development of gene therapies to treat ornithine transcarbamylase deficiency, glycogen storage disease type Ia, CDKL5 deficiency, hemophilia A, and Wilson disease.

In a separate agreement, Ultragenyx entered into a strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo for its 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. Ultragenyx has also developed a proprietary HEK293 transient transfection system for AAV manufacture which is also part of the collaboration.

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 companies 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 Kakkis.

Daiichi Sankyo will be responsible for the manufacture, development, and commercialization of products manufactured with the Ultragenyx technology, with Ultragenyx receiving an option to co-develop and co-commercialize rare disease products at the IND stage. Ultragenyx will also provide strategic consultation to Daiichi Sankyo on the development of both AAV-based gene therapy products and other products for rare diseases.

“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, executive officer and 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.”

Under the terms of their agreement, Daiichi Sankyo will make an upfront payment of $125 million and will purchase $75 million of Ultragenyx common stock at a price of approximately $60 per share. Daiichi Sankyo will pay an additional $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. Daiichi Sankyo will also reimburse Ultragenyx for all costs associated with the transfer of the manufacturing technology.