Research and Development Strategy for Future Embryonic Stem Cell-Based Therapy in Japan.

Area of Science:

• Regenerative Medicine
• Stem Cell Biology
• Translational Research

Background:

• Human embryonic stem cells (ESCs) hold promise for regenerative medicine.
• Previous ESC derivation in Japan utilized animal feeder cells, posing potential risks.
• Establishing clinical-grade ESCs is crucial for therapeutic applications.

Purpose of the Study:

• To review the progress of human ESC-based regenerative medical products in Japan.
• To highlight the development of a novel, clinically relevant ESC derivation and cultivation system.
• To detail the regulatory steps for advancing ESC-based therapies.

Main Methods:

• Development of a novel xeno-free cultivation system for human ESCs.
• Establishment of twelve clinically relevant human ESC lines (KhES and SEES series).
• Submission of an investigative new drug application for ESC-based products.

Main Results:

• A xeno-free system was successfully established, minimizing animal product exposure.
• Twelve human ESC lines are now available on a clinically relevant platform.
• An investigative new drug application was submitted for treating hyperammonemia.

Conclusions:

• Japan has made significant progress in developing human ESC-based regenerative therapies.
• The established xeno-free system facilitates the translation of ESC research into clinical practice.
• Multiple ESC-based products are in development for intractable and rare disorders in Japan.