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Our Initiatives to Create Innovation

Case 1 Accelerating R&D in Ophthalmology

We will establish a cell therapy franchise in ophthalmology by
pursuing “Best Science, Best Talent, Best Place.”

Yoshitsugu Shitaka, Ph.D.
President, Astellas Institute for Regenerative Medicine (AIRM)

Portrait of Yoshitsugu Shitaka, Ph.D

Strong Start for AIRM Leveraging Both Companies’ Complementary Technologies, Experience, and Financial Bases

There remains many diseases of the eye with no therapeutic options. In particular, we are still waiting for groundbreaking drugs to treat conditions that lead to vision impairment. In addition to developing small molecule VAP-1 inhibitor ASP8232* in-house, Astellas is researching new technologies to support cell and gene therapies in this area, including collaboration with Harvard Medical School, a global front-runner in gene therapies for ophthalmic diseases.
Many conditions that can lead to loss of sight involve the degeneration or loss of the cells responsible for visual function. There is a rational case for developing cell therapies to treat such conditions because (1) function can be restored with replacement of the affected tissue, (2) fairly few cells are needed for transplanting, (3) cells can be transplanted directly to diseased tissue, (4) rejection is rare, and (5) precise clinical trial endpoints can be defined.
We acquired Ocata Therapeutics, Inc. (Ocata), one of the leaders in cell therapy advancing ophthalmic R&D, in order to advance research and development largely with their capabilities. They have world-class technology and expertise in ways of sourcing differentiated cells from pluripotent stem cells. Ocata’s researchers lead the world in this field and they also have clinical development experience in cell therapy. The Company’s name was changed to the Astellas Institute for Regenerative Medicine after the acquisition. Following my appointment as President of AIRM and after only a few months in the job, I am convinced of the huge potential of these R&D activities.

Most of the former key Ocata staff have stayed at AIRM and are highly motivated to continue the R&D programs. One reason for this is the extremely good fit between Astellas and Ocata in terms of complementary functions. Astellas’ quality control, cross-divisional project management expertise, and understanding of diseases and conditions prove valuable in advancing current pipeline projects. The global network and experience of Astellas will also be essential in advancing the projects currently at the clinical trial stage to commercialization. In that sense, many have commented that Ocata and Astellas joined forces at an extremely opportune moment.
* Currently in Phase II for diabetic macular edema.

Positioning of AIRM in Strategic Plan 2015−2017


Turning Challenge into Opportunity: First Aim to Create Cell Therapy Franchise in Ophthalmology

Since we are the front-runner, the absence of useful precedents means we have to take projects forward based on internal debate over study protocols, manufacturing methods, and quality standards. We have a shared understanding that we must change our challenges into opportunities, targeting a world-first treatment by accelerating R&D projects as much as possible. The goal of AIRM is to advance multiple R&D projects as far as the clinical development stage within just a few years.
From a longer-term perspective, AIRM also aims to take on the challenge of regenerative medicine. In line with this, since becoming AIRM, we have resumed a number of projects with potential to broaden the project scope beyond our traditional focus on ophthalmology. AIRM is also actively hiring. Under the leadership of Dr. Lanza, AIRM’s talented personnel, technologies, and R&D projects in the field of regenerative medicine are attracting interest from many outstanding scientists who would like to join our team. Going forward, we expect to form a suitable team to work at the forefront of Astellas’ research in this area. Based on the “Best Science, Best Talent, Best Place” notion, Astellas will pursue cutting-edge medical science through the talents, technology and clinical development experience of AIRM, including regulatory expertise. We will leverage the research network comprising AIRM located in the Boston area where research in regenerative medicine thrives and the Group’s Regenerative Medicine Labs in Japan. The overall aim is to establish a cell therapy franchise in ophthalmology and to build a leading position in cell therapy.

Establishing a Presence in Ophthalmology


Adding the knowledge and understanding of Astellas’ talented researchers has made the company far stronger.

Ocata’s vision was to be a leading ophthalmology company using its proprietary regenerative medicine platform to cure loss of vision. Astellas shares this same goal. Our pipeline includes several therapies with great clinical promise, notably the use of retinal pigment epithelium (RPE) cells for treating dry age-related macular degeneration (AMD). The creation of AIRM has made our organization stronger and enabled us to expand the scope of our research to include programs in other areas. Adding the knowledge and understanding of Astellas’ talented researchers, we will not only be able to advance our current projects in ophthalmology, but also help usher in a revolution by applying regenerative medicine across multiple fields.

Portrait of Robert Lanza, M.D.

Robert Lanza, M.D.
Head of Astellas Global Regenerative Medicine and Chief Scientific Officer AIRM

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