Astellas Innovation Center in South San Francisco
In an exclusive interview with Drug Discovery & Development, Astellas Pharma CEO Naoki Okamura and Chief Scientific Officer Yoshitsugu (Yoshi) Shitaka shared details about its new $90 million, 154,000-square-foot biotech facility in South San Francisco. The new Astellas facility will serve as a central hub for Astellas’ Bay Area employees and be pivotal for the company’s cell and gene therapy research and development efforts.
The news comes as regulatory traction builds for cell and gene therapies. In 2023, a record number of gene therapies won FDA approval, and the momentum continues into 2024. “For our company and also as an industry, more and more gene therapy programs will be approved in the coming 4-5 years,” predicted Shitaka.
New Astellas’ $90 million Bay Area hub to fuels CGT ambitions
Naoki Okamura
While the new South San Francisco outpost will be a pillar in the company’s cell and gene therapy strategy, including the development efforts of AT845, an investigational adeno-associated virus (AAV) gene replacement therapy, for the treatment of Late-Onset Pompe Disease (LOPD), it also reflects Astellas’ commitment to global collaboration. “We have a completely global operation,” explained Okamura. “Sometimes my direct reports are in Chicago, his direct reports are in Tokyo and London. We no longer think of ourselves as having a U.S. operation here and a European operation there. We have a completely global operation.”
The new South San Francisco facility further supports this global collaborative vision by uniting researchers from diverse locations and disciplines under one roof. “Putting them all together is potentially the source of innovation,” Okamura explained. “There could be connections between totally unexpected combinations of biology and modality.” Shitaka provided an example of this cross-pollination in action: “We are sending researchers from Tsukuba [in Japan], to South San Francisco or to Boston and vice versa. The researchers themselves are sometimes stuck to a certain location, but we have the way to really mix up the people across the technology platforms and different regions. We encourage them to speak directly to each other.”
Disease biology at the core
Yoshitsugu Shitaka, Ph.D.
The company’s emphasis on expertise is also apparent in its approach to selecting therapeutic targets, which starts with a deep understanding of disease biology. The new South San Francisco facility embodies Astellas’ approach of starting with a disease biology research question. “We are not starting from the therapeutic area, but we start from the biology and trying to find out the best modality to address this biology,” Okamura explained. “And when we have this combination, we start thinking what would be the best patient population to benefit from this combination of biology and modality.”
Astellas has a growing U.S., and global, footprint
Astellas, which counts itself as one of the largest Japan-based pharma companies, has a variety of U.S. locations. While its U.S. headquarters is in Northbrook, Illinois, its government affairs operations are in Washington D.C., and biomedical innovation hubs in Westborough, Massachusetts, and Cambridge, Massachusetts. U.S. affiliates Universal Cells and Xyphos Biosciences are based in Seattle and San Francisco, respectively. Outside of the U.S. and Japan, Astellas has a string of facilities throughout the world, including in an expanding manufacturing footprint in Ireland.
The significant U.S. footprint is a key component of Astellas’ global product launch strategy. “For the U.S. market, our basic approach is to prioritize launching our products first in the United States,” Shitaka said. With the U.S. market continuing to drive the bulk of many firms’ revenue, it is a core focus for many pharma firms’ commercialization efforts, including Astellas. “Regardless of where the product is developed—whether in the U.S., Japan, or our Tsukuba facilities—basically, our strategy is to launch our products in the U.S. first.”
Astellas aims to continue revamping R&D with AI and robotics
Beyond its global product launch strategy, Astellas is also focusing on technological innovations to bolster its R&D efforts. Two growing cornerstones for Astellas’ R&D efforts are machine learning and robotics. Astellas is tapping AI and machine learning across its research areas, including small molecules, biologics, cell therapy, and gene therapy.
AI is not a new priority for Astellas. “We are working on small molecule biologics, cell and gene therapy. In each area, we have already used AI,” Shitaka said. “For example, in small molecule, we already have a good track record using AI.” Shitaka note that, on average, it takes about one to two years to fully optimize a compound traditionally. “But by using AI/machine learning, we shortened that time period 70%.”
In biologics, Astellas is using AI to help design protein sequences. “In cell therapy, we use AI to predict key transcription factors for differentiation,” Shitaka added. In cell therapy, Astellas is collaborating with a partner to use AI to predict key transcription factors for differentiation. “So on all fronts, we have used AI already,” Shitaka said.
Astellas has a strategic partnership with the University of Tsukuba to accelerate innovative drug discovery research using AI and digital technologies. Shitaka notes that the company broadly collaborates with external AI experts and has formed an alliance with NVIDIA to access more GPU capacity for deep learning.
Blurred lines between AI and robotics
From a simple perspective, AI and robotics are a natural combination, with the former serving as a brain and the latter a body when the two are combined. “AI and Robotics have a very close relationship,” Shitaka said. Like other drug developers, Astellas generates massive data sets that can be used as training data for machine learning. In the domain of robotics, machines excel at repeating precise movements — “much better than experienced operators,” Shitaka said. “In cell therapy, the process is the product. The idea is that a slight procedure or movement change will affect the results and change the property of the final cell product. In that sense, we think it’s very important to incorporate robotics.”
One of Astallas’ core robotic projects is the Mahol-A-Ba, which automates cell culture and differentiation processes. This advanced automation allows experiments that would typically take a human researcher a month to complete to be finished within a single day. While the robot is now limited to its Tsukuba facility, Astellas plans to export Mahol-A-Ba robots to its facilities.
The video below shows the cellular drug discovery robot, Mahol-A-Ba, in action.
From collaboration to more active contribution
In addition to robotics and AI, Astellas also recognizes the importance of external partnerships in driving innovation. The company has formed strategic partnerships with several companies and institutions to advance its research and development efforts in various therapeutic areas. These collaborations include alliances with 4D Molecular Therapeutics for gene therapies in ophthalmology, CytomX Therapeutics and Sutro Biopharma for oncology treatments, Mass General Brigham for translational research in oncology, rare diseases and cell/gene therapies, Poseida Therapeutics for allogeneic CAR-T cell therapies in solid tumors, and Kelonia for in vivo delivery to create CAR cells, with targets not disclosed.
“So this new facility is the natural consequence that I mentioned, starting with partnerships, and then consolidating. Now this facility can work as a hub to explore even more partnerships and, as the CEO said, start contributing to the ecosystem rather than just benefiting from it,” Shitaka explained. “We have first consolidated our efforts, but now we start really going out to explore and even contribute to the South San Francisco ecosystem. So that’s the change from just benefiting from them to really contributing to them.”
Filed Under: Cell & gene therapy, Data science, machine learning and AI
