Kyoto University: Platelets are made from iPS cells and administered: Venture ‘Megakaryon’
-Mass production of platelets necessary for hemostasis-
-Clinical trials will start by the end of the year-
Kyoto University Venture:
It produces blood components called platelets from iPS cells.
A clinical trial plan for a treatment that administers this to patients has begun.
“Megakaryon”:
Kyoto City’s bio-venture company “Megakaryon” announced on April 26th.
Since the investigation by the Japanese government agency has been completed
After undergoing medical institution examination,
We want to start a clinical trial by the end of the year.
Platelet production from iPS cells:
This company produces platelets from iPS cells, which is one of the components of blood and is necessary for hemostasis.
We are in the process of conducting a clinical trial plan to obtain national approval for the treatment of patients with “thrombocytopenia.”
Kyoto University provides iPS cells:
In the clinical trial plan
From third-party iPS cells
Produces platelets,
Administered to 10 adult men and women
We will spend a year checking the safety and effectiveness.
Mass production of platelets:
Traditionally, platelets used for treatment have relied on blood donations.
If it can be produced from iPS cells, mass production will be possible.
Medical | NHK News
https://www3.nhk.or.jp/news/html/20210426/k10012997531000.html
Megakaryon Corporation announces the completion of 30-day review of Clinical trial notification submitted for human iPS cell-derived HLA homozygous platelets (MEG-002).
26th April 2021
Overview
Megakaryon Corporation
today announces the completion of 30-day review of the clinical trial notification submitted for human iPS cell-derived HLA homozygous platelets (Development Code: MEG-002)
by Japan’s Pharmaceutical and Medical Devices Agency (PMDA).
Megakaryon Corporation
has planned this trial MEG-002 in collaboration with Kyoto University Hospital,
Center for iPS Cell Research and Application, Kyoto University(CiRA) and CiRA Foundation(CiRA_F).
We will continue to work closely with related parties and work diligently toward conducting the clinical trial.
Background
The Department of Hematology, Kyoto University Hospital (Professor Akifumi Takaori)and CiRA (Professor Koji Eto)
have conducted clinical research for the transfer of autologous iPS cell-derived platelets to a thrombocytopenia patient to verify the safety of iPS cell-derived platelet preparations.
The clinical trial
announced today is for an allogeneic transplant, which will enable industrialization of iPS cell-derived platelet and subsequent availability to a large number of people.
Summary of the clinical trial
In the clinical trial, we will confirm the safety and estimate the efficacy of MEG-002 to thrombocytopenia patients.
MEG-002 is prepared from an iPS cell provided by CiRA (currently CiRA_F) and consists of platelets with the HLA type most common in Japanese people.
The technology for producing platelets from iPS cells 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, Kyoto University Hospital using product manufactured by CiRA_F.
Support for the clinical trial
The practical application of human iPS cell-derived HLA homozygous platelets
will be implemented with the support of the 5th Cyclic Innovation for Clinical Empowerment (CiCLE) program of Japan Agency for Medical Research and Development (AMED).
Megakaryon Corporation
was established in 2011 with the aim of utilizing technologies invented by Professor Koji Eto and others for producing platelets from human iPS cells for clinical application.
By developing large-scale manufacturing of human iPS-derived platelets with no risk of infection, we aim to supply platelets to medical facilities around the world.
Human iPS-derived platelets
Human iPS cell-derived platelets are produced by maturing megakaryocytes cultured from a master cell bank (MCB).
MCB is made from cryopreservable immortalized megakaryocyte precursor cells obtained by introducing three genes into hematopoietic precursor cells differentiated from human iPS cells.
Platelets, Thrombocytopenia
Platelets, also known as thrombocytes, are major blood components that play a crucial role in hemostasis.
Upon endothelial damage, platelets are activated leading to adhesion and aggregation at the wound site, thereby stopping bleeding.
Thrombocytopenia is a condition in which the number of platelets in the blood is low.
If the platelet count drops below a certain level or if there is a high risk of bleeding, treatment with blood transfusion platelet preparations is given.
http://www.megakaryon.com/en/dataroom/pdf/mega_release_20210426_en.pdf