From The Editor | December 13, 2021

Astellas' Active Strategy to Gain Access to New Technologies


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1

Teamwork partnership GettyImages-1196912174

Astellas Institute for Regenerative Medicine (AIRM) has been methodically building, investing, and partnering in cell and gene therapy for quite some time. In the field of cell therapies, the company’s collaboration and M&A deals span stem cell therapy, allogeneic cell technology, and CAR cell therapy. AIRM’s President, Hide Goto, Ph.D., and I discussed the collaboration and M&A deals as well as its innovation hubs in detail. Read on to understand how AIRM benefits from active and open collaboration and how other biotechs can ­­do the same.

Explain how collaboration and M&A deals will progress Astellas’ work in each of the three areas: Stem cell therapy; allogeneic cell technology; and CAR cell therapy.

Goto: We have been building, investing, and partnering in the cell and gene therapies fields for several years; these areas represent those which Astellas has chosen for long-term opportunities to invest in scientific research with the potential for major therapeutic breakthroughs. In cell therapies, this vision has come to fruition as our strategic initiatives have brought together capabilities that represent one of the most comprehensive regenerative medicine centers in the industry – the Astellas Institute for Regenerative Medicine (AIRM). AIRM serves as a hub for our range of synergistic cell therapy expertise across multiple locations.

The foundation of AIRM began in February 2016, when Astellas acquired Ocata Therapeutics, one of the earliest biotech companies developing stem cell therapies under the leadership of stem cell pioneer, Dr. Robert Lanza. As cell therapy science continued to advance at rapid speed, Astellas methodically put together complementary skills and scientific innovation for world class stem cell therapies. Our vision remained steadfast: to advance regenerative medicines to be off-the-shelf cell therapies, designed to reach many diseases, and bring value to patients.

In February 2018, Astellas acquired Universal Cells in Seattle with unique technology with the potential to produce pluripotent stem cells that have lower immunological rejection potential to create allogeneic therapies, or off-the-shelf cell therapies, which could possibly treat, and potentially even cure, a wide range of diseases. Then, in December 2019, Astellas acquired Xyphos Biosciences in San Francisco, with a proprietary approach for “convertible CARs”, which use synthetic biology methods to adapt cell therapies for potential increases in flexibility, control, and  safety. This could result in techniques for state-of-the-art CAR-T cells as well as other immune cell and stem cell applications.

Overall, our goal is to create valuable new therapeutic solutions by bringing together these cutting-edge companies and technologies in the promising field of cell therapy.

Astellas has a guiding principle for active and open collaboration in cell and gene therapy as a core way to tap into the proprietary technologies and unique scientific know-how of emerging biotech companies. What is the guiding principle for active and open collaboration? What makes it different and distinct from other biotechs? And, what benefits does active and open collaboration afford Astellas and what can other biotechs learn from this?

Goto: We believe that pursuit of the boldest new fields of science that have breakthrough potential requires collaboration. Astellas has been collaborating with academic researchers and a range of companies which we believe have leading-edge science and capabilities. We seek out collaborations with a variety of partners to translate innovative science into therapeutic solutions in areas with high unmet medical needs and value for patients. When it comes to partnering, we want to maintain a collective focus on delivering value to patients.

As a guiding principle, Astellas is committed to working collaboratively with key opinion leaders, academics, and new and evolving biotech companies to accelerate the discovery and development of new gene and cell therapeutics. We can offer Astellas’ global expertise and end to end resources to our partners so that together we can successfully develop new medicines in these areas.

With the remarkable breadth of advances in science, we are continuously on the search to identify new opportunities that will impact and drive our drug development pipeline today and for the long-term future including such areas as gene and cell therapies. At Astellas, open innovation is a core part of our culture and critical as a component of our long-term strategy and success.

We allow strong science teams from our partnerships and acquisitions to maintain their independence, so that that those closest to the science drive the progress. Scientists who work with Astellas have the independence to innovate with local decision-making by those who are closest to the science and discoveries they uncover.

Our success relies on the partnerships we have built to date as well new collaborations and acquisitions to ensure we have the expertise and right science, so that our drug development teams can achieve and deliver the breakthroughs necessary for new therapeutic developments.

Astellas has established innovation hubs for cell therapy in Boston and San Francisco/West Coast. Explain the innovation hubs in detail.

Goto: Astellas has established hubs for advancing the latest drug discovery technologies in key biotech ecosystems, and this includes the cell therapy capabilities we have built on the east coast and west coast of the U.S. By being in these locations, we can work with leading academic researchers, incubators, biotech companies and venture capitalists to form collaborations that bring together synergistic resources and technologies.

In the Boston area, Astellas has created AIRM, which has grown to become one of the most comprehensive regenerative medicine centers in the industry, embodying our commitment to developing cell therapies. The 262,000 square-foot facility is home to more than 150 researchers, houses one of the industry’s largest stem cell libraries, and uses advanced manufacturing.

On the West Coast, we have brought together several important technologies that bring state-of-the-art technologies to our development of cell therapies. Allogeneic cell therapy technology has been developed and continues to advance with our Universal Cells team in Seattle, and this capability is fundamental to our work in creating off-the-shelf cell therapies for broad applications. Another key cell therapy capability is our CAR-T technology for cancer-fighting cell therapies, which is led by our Xyphos team in San Francisco.  

The Institute is creating a center of excellence – with capabilities spanning pluripotent cell development, cell differentiation process development, and manufacturing – and taking an important step forward to re-defining this class of medicines to treat a broad range of diseases. AIRM’s researchers are currently investigating pluripotent stem cell-derived therapies that have the potential to rejuvenate, regenerate and replace damaged tissues, or be the starting material for allogeneic cell therapies.

We have assembled capabilities across a range of technologies, expert teams, and locations because we believe the complexity of cell therapies requires a combination of new scientific and R&D approaches. At each of our locations, our teams have active cell therapy programs that are advancing toward clinical trials, and we are excited about the progress ahead.

Explain Astellas’ pipeline strategy, highlighting some of the early programs for advancing cell therapies.

Goto: In creating cell therapy products, we focus on advancing solutions where our scientists have translated cell engineering technologies into therapeutics and connecting them to diseases where we can address significant unmet needs of patients.

I’ll highlight two prime examples. In the area of ocular diseases, we are applying the next-generation modality of cell therapy to potentially treat back-of-the-eye diseases at high risk of blindness. With cell therapy, we are pursuing a new therapeutic approach for eye diseases that lack treatment options or have been difficult to address with existing therapeutic approaches. Among the ocular diseases we are pursuing are acute macular degeneration, glaucoma, and retinitis pigmentosa.

Another key area of focus for us is innovative cell therapies for cancer. For example, we are developing CAR-NK cell therapies and CAR-T cell therapies, which are new classes of cell therapies that are changing the treatment landscape for cancer. These CAR cell therapies require a suite of advanced technologies to design and manufacture them with properties of safety, tumor targeting, and cytotoxic activity, as well as the ability to be engineered for off-the-shelf production with an efficient manufacturing process. Allogeneic CAR cell therapies offer unprecedented potential for treating many types of cancers, and yet challenges remain. With the multiple advanced technologies and the collaboration of our cell therapy teams at Astellas, we aim to overcome these challenges with CAR-T and CAR-NK cell therapy products for both hematologic and solid tumors.

Following these two leading areas, we are trying to expand our target therapeutic areas, like autoimmune and vascular related diseases, based on our solid scientific and technological knowledge.  We are continuously striving to deliver value to patients who are suffering from incurable diseases.

What processes are you putting in place to get cell therapies to patients?

Goto: At Astellas, we are continuing to build a collection of cell therapy technologies and capabilities, combined with the commercialization skills of our global organization, to advance this new class of cell therapy medicines to patients. The path from lab to clinic to patient for cell therapies presents a unique set of drug development and commercialization challenges that are different from typical pharmaceuticals and biologics.

Novel cell therapy approaches bring new scientific and R&D complexities that are different from small molecules or antibodies. From the very outset, for certain cell therapies, there can be challenges with production of cell lines, which can limit the quality and quantity of raw materials. Even after achieving clinical proof of concept, there are still myriad additional hurdles ahead to produce a cell therapy, including:

  • Manufacturing: Achieving consistent and controlled production is one of the most unique and challenging components of creating a cell therapy. By their very nature as living cells, cell therapies are complex and variable, and can present challenges in product testing and characterization required for a safe and robust manufacturing process that follows Good Manufacturing Practice (GMP) principles.
  • Supply Chain: Therapies from living cells, being highly sensitive to time and temperature, can create supply chain challenges and, in many cases, require new technology solutions and monitoring to ensure end-to-end quality and traceability. New models for distribution networks are emerging and for this class of medicines to become mainstream, the healthcare ecosystem must adapt.
  • Pricing and Access: The prospect of drugs that enable the body to heal itself with a single treatment requires an entirely new model for pricing and access. The first wave of approved cell therapies is paving the way for important discussions about a treatment’s value — including pricing and reimbursement models — so that patients can access these potentially curative medicines.

At Astellas, we recognize that it's important to bring together technologies across the value chain from product design to manufacturing to patient access and delivery in order to bring these potentially transformative therapies to patients.