京都大学：血小板由iPS细胞制成，并经管理：Venture’Megakaryon’
-大量生产止血所需的血小板-
-临床试验将于今年年底开始-
京都大学创业：
它从iPS细胞产生称为血小板的血液成分。
一项针对患者进行治疗的临床试验计划已经开始。
“超级英雄”：
京都市的生物冒险公司“ Megakaryon”于4月26日宣布。
由于日本政府机构的调查已经完成
经过医疗机构检查后，
我想在今年年底开始一项临床试验。
iPS细胞产生的血小板：
该公司从iPS细胞生产血小板，iPS细胞是血液的成分之一，是止血所必需的。
我们正在执行一项临床试验计划，以获得治疗血小板减少症患者的国家认可。
京都大学提供iPS单元：
在临床试验计划中
来自第三方iPS单元
产生血小板
适用于10名成年男性和女性
我们将花费一年的时间检查安全性和有效性。
大量生产血小板：
传统上，用于治疗的血小板依赖于献血。
如果可以从iPS电池生产，则有可能批量生产。
医疗| NHK新闻
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