Astellas Pharma Inc.’s U.S.-based regenerative medicine center has agreed to pay Seattle-based Universal Cells Inc. up to $124 million for global rights to use its Universal Donor Cell technology in a new cell therapy for an undisclosed indication. Universal is eligible to receive up to $9 million in upfront and research milestones from Astellas and up to $115 million more for research, clinical, and regulatory milestones, plus royalties.
Yoshitsugu Shitaka, president of the Astellas Institute for Regenerative Medicine, told BioWorld that Universal’s technology may allow Astellas to create cell therapies that are immune-rejection free, so they can be administered to any recipient without need for human leukocyte antigen (HLA) matching. “Essentially, this means that cell therapy products derived from one starting cell line (ES cell or iPS cell), edited by ‘Universal Donor Cell’ technology, can then be applied to any recipients, which may enhance efficiencies in our regenerative medicines business,” he said.
The deal is part of a growing stable of partnerships producing meaningful R&D funding for Universal, which has otherwise bootstrapped its growth on the Seattle waterfront using little more than seed and NIH funding. Earlier this month, in a separate deal, it agreed to create induced pluripotent stem cell lines for Bayer AG-backed Bluerock Therapeutics. And before that came deals with Healios K.K. in macular degeneration and Adaptimmune Therapeutics plc for the creation of new allogeneic T-cell therapies. (See BioWorld Today, Dec. 2, 2015.)
Universal’s key intellectual property covers the use of any gene editing technology to modify pluripotent stem cells to control how they interact with the immune system through HLA molecules. Through homologous recombination, expression of polymorphic HLA molecules by the cells is prevented, thus removing the key cause of donor tissue rejection. That means that “you don’t have to put the patients on regimens of immunosuppressive drugs,” Greg Block, Universal Cells’ vice president of corporate development, told BioWorld. “You don’t have to worry about the development of anti-HLA antibodies, which complicate transplantations or re-transplantations. That’s the holy grail of regenerative medicine that we’re working toward here,” he said.
A key element of the company’s success in attracting large partners and creating deals of the magnitude of Astellas, he said, has been the in-house capability it has built for manufacturing edited cells that can go into the clinic. Gene editing is a hard and complex procedure that requires massive investments in time, training and quality assurance, he explained. Now, with the investments Universal Cells has made “we can make a very compelling case that what we’ve said we’re doing is what we’ve done,” he said.
A long lineage
The Astellas Institute for Regenerative Medicine was established in May 2016 following the Tokyo-based company’s acquisition of Ocata Therapeutics Inc. Headquartered in Marlborough, Mass., and supported by a research team in Tsukuba, Japan, it inherits a long history of steady slow scientific progress first set in motion with the founding in 1994 of Ocata predecessor Advanced Cell Technology, Inc. (See BioWorld Today, Nov. 11, 2015.)
Astellas has prioritized ophthalmology as part of its strategy for sustainable growth, focusing on treatments for disorders of the posterior segment of the eye where no standard drug treatments are currently available. Retinitis pigmentosa, dry age-related macular degeneration, Stargardt’s macular degeneration and diabetic macular edema have all been areas of interest.
However, that doesn’t necessarily mean its work with Universal will be an ophthalmologic indication. “Regenerative medicine has the potential to change the thinking about disease, aging and even the practice of medicine itself,” the company points out on its website. With that mindset, its licensing of a technology that could bring cell therapy to the masses, makes perfect sense.