Megakaryon Corporation Initiates First-in-Human Clinical Trial of Allogenic Human iPSC-Derived HLA Homozygous Platelets (MEG-002)

KYOTO, Japan--()--Megakaryon Corporation (hereinafter, Megakaryon) today announces its clinical trial of allogenic human iPSC-derived HLA homozygous platelets (Development Code: MEG-002) in collaboration with Kyoto University Hospital, Center for iPS Cell Research and Application, Kyoto University (CiRA) and CiRA Foundation (CiRA_F).
Initial administration to the first subject was successful and with no reported adverse events.


The Department of Hematology, Kyoto University Hospital (Professor Akifumi Takaori-Kondo, M.D., Ph.D.) and CiRA (Professor Koji Eto M.D., Ph.D.) have conducted clinical research for the transfer of autologous iPSC-derived platelets to a thrombocytopenia patient to verify the safety of iPSC-derived platelet preparations. The clinical trial announced today is for a study using allogenic human iPSC-derived HLA homozygous platelets developed by Megakaryon which will enable industrialization of iPSC-derived platelets and their subsequent availability to a large number of patients.

About the first administration of investigational product


Kyoto University Hospital

Dosing date:

April 2022

Coordinating investigator:

Akifumi Takaori-Kondo, M.D., Ph.D., Professor of Hematology and Oncology

Principal investigator:

Junya Kanda, M.D., Ph.D., Associate Professor of Hematology and Oncology

Clinical trial products:

Allogenic human iPSC-derived HLA homozygous platelets (MEG-002)


3 units (0.6x10^11 platelets)

Manufacturing location:

Facility for iPS Cell Therapy (FiT) of CiRA_F

Summary of the clinical trial

During the clinical trial, Megakaryon will assess the safety and estimate the efficacy of MEG-002 for patients with thrombocytopenia. MEG-002 is prepared from an iPSC provided by CiRA (currently CiRA_F) and consists of platelets with the most common HLA haplotype among the Japanese population. The technology for producing platelets from iPSCs invented by Professor Koji Eto is used for the development of MEG-002. The clinical trial will be conducted at multiple medical institutions, including the Department of Hematology and Oncology, Kyoto University Hospital using a product manufactured by CiRA_F.

Title of the clinical trial

Exploratory clinical study on the tolerability, safety and efficacy of iPS cell-derived platelets (MEG-002) in patients with thrombocytopenia

Purpose of the clinical trial

Tolerability, safety and efficacy of a single dose of MEG-002 in thrombocytopenia patients.

Design of the clinical trial

Phase 1/2 study:

Open-label, uncontrolled study / single-dose study

Dose (1):

3 units (0.6x10^11 platelets)

Dose (2):

10 units (2x10^11 platelets)

Primary endpoint


Incidence of adverse events and side effects


Corrected count increment (CCI)

Period of the clinical trial

July 2021 – August 2022 (Planned to be extended)

For more information at Japan Registry of Clinical Trials (jRCT).

Megakaryon Corporation

Head office: Shimogyo-ku, Kyoto, Japan
President: Kenichi Akamatsu


Megakaryon Corporation was established in 2011 with the aim of utilizing technologies invented by Professor Koji Eto and others for producing platelets from human iPSCs for clinical application. By developing large-scale manufacturing of human iPSC-derived platelets with no risk of infection, we aim to supply platelets to medical facilities around the world.


Media Contact:
Megakaryon Corporation
Tsuyoshi Ayabe


Media Contact:
Megakaryon Corporation
Tsuyoshi Ayabe