Bringing New Solutions into View for Cell and Gene Therapies: Masahide Goto, President, AIRM

Masahide (Hide) Goto is President, Astellas Institute for Regenerative Medicine (AIRM). Headquartered in Westborough, MA, AIRM is an indirect, wholly owned subsidiary of Astellas and serves as the company’s global hub for regenerative medicine and cell therapy research and manufacturing in ophthalmology and other therapeutic areas that have few or no available treatment options.

Astellas has identified focus areas with significant upside potential for patients including cell and gene therapies. In this interview, Goto describes the company’s initiatives in these areas.

Pharm Exec: What role does collaboration and partnering play in your cell and gene therapy strategy?

Hide Goto

Hide Goto: With the remarkable breadth of advances in science, we are continuously on the search to identify new opportunities that will impact and drive our cell and gene therapy development pipelines today and for the long term. To be successful in these rapidly evolving areas, Astellas has a philosophy of continually accessing new and evolving science and technologies. We believe the foundation for success is our strong network of leaders in the scientific community with whom we work closely to identify and create the necessary win-win collaborations and partnerships.

Our collaborations with key opinion leaders, academics, and new and evolving biotechnology companies allow us to accelerate the discovery and development of new gene and cell therapies. Undoubtedly, success relies on the partnerships we have built to date, as well as new collaborations and acquisitions that ensure we have the expertise and strong science, so that our drug development teams can achieve and deliver new solutions necessary for these complex therapeutics.

We realize that the future of our pipeline depends on collaborations; they deliver value and allow us to translate innovative sciences into new gene and cell therapies to address high unmet medical needs for patients. An essential component of these collaborations, and a core part of our culture, is open innovation. Through our partnerships we have built and committed to continually creating strong independent drug development teams. These teams are the drivers of our future products. We have our sights set on building and expanding our knowledge and capabilities so that these scientific teams can be successful in accelerating the progression of new therapeutics from the bench to the clinic to patients.

Tell me about your areas of expertise in cell therapy.

Astellas has established cell therapy hubs on the east and west coasts of the US. These locations were selected as they allow us to be close to and collaborate with leading academic researchers, incubators, biotech companies and venture capitalists.

With more than 150 researchers based just outside of Boston, the Astellas Institute for Regenerative Medicine (AIRM) has grown to become one of the most comprehensive regenerative medicine centers in the industry. The Institute is establishing 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.

On the west coast, we have brought together state-of-the-art technologies for cell therapy development. 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.Our chimeric antigen receptor (CAR) technology for battling cancer is led by our Xyphos team in San Francisco.

What are you doing in the emerging area of CAR therapies for cancer?

We are developing both CAR-T and CAR-NK cell therapies. These 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 and partners, we aim to overcome these challenges with CAR-T and CAR-NK cell therapy products for both hematologic and solid tumors.

Based on our solid scientific and technological knowledge, we are also looking to expand our target therapeutic areas into autoimmune and vascular related diseases. Across these areas, we are continuously striving to deliver value to patients who are suffering from incurable diseases.

What are some of the key challenges related to the delivery of cell and gene therapies?

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. The entire value chain is important to consider in the delivery of cell and includes efficient manufacturing, delivering the product to the patient, and ensuring patient access for these specialized therapies.

Novel cell therapy approaches, for example, 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 a specific cell population, 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 cold chain capabilities, new technology solutions and monitoring to ensure end-to-end quality. 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 and gene 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.

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