Focus Area Approach

Initiatives from Research to POC*

* Proof of Concept (POC): Verification of clinical efficacy

Focus Area Approach


Advancing Cell Therapy

Astellas began its drug discovery initiatives through an approach combining the biology of regeneration and the modality of cell therapy, applying it to explore treatments in the field of ophthalmic diseases. We will expand this approach to other diseases in the future.

Commenced R&D in Ophthalmology

If cells abandon their original functions resulting in diseases, administering cells could be a treatment. In focusing on regeneration as biology closely related to pathophysiology and treatment, Astellas has commenced R&D based on the new modality of cell therapy.

Astellas is first trying to apply this approach to the field of ophthalmology. In theory, the field of ophthalmology is suitable for cell therapy due to three main reasons: (1) The loss or dysfunction of cells is clearly connected with the resulting diseases, (2) The small size of eye tissue requires transfusion of only a relatively small number of cells, and (3) The intraocular region is isolated from immune reactions of the body. Notably, there are significant unmet medical needs for the treatment of diseases in the posterior segment of the eye, which can lead to vision loss. Currently, Astellas is developing ASP7317 for dry age-related macular degeneration (AMD) and Stargardt’s macular degeneration. ASP7317 is a project derived from Ocata Therapeutics, Inc., which was acquired in 2015. ASP7317 is currently in the Phase 2 stage.

Introducing New Modalities to Expand Target Diseases

Ocata Therapeutics was one of the world’s frontrunners in the fundamental technology for establishing fully differentiated cells from pluripotent stem cells. Ocata Therapeutics has now changed its name to the Astellas Institute for Regenerative Medicine (AIRM) with further strengthened capabilities. We also plan to invest in manufacturing facilities at AIRM in anticipation of the commercialization of cell therapy.

Moving forward, Astellas will also expand its approach of combining regeneration and cell therapy to fields beyond ophthalmology. In the course of expanding into other therapeutic areas, one challenge will be to suppress the immunological rejection of transplanted cells. To resolve this issue, Astellas acquired Universal Cells, Inc. in February 2018. Universal Cells’ proprietary Universal Donor Cell technology uses genome editing to prevent the expression of polymorphic human leukocyte antigen (HLA) molecules, thereby suppressing immune reactions caused by mismatched HLA at transplantation. By combining the technologies of AIRM and Universal Cells, Astellas will expand the scope of cell therapy from eye diseases to a variety of other diseases.

Expansion of the Focus Area Approach

Chart:Expansion of the Focus Area ApproachChart:Expansion of the Focus Area Approach
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