CiRA Foundation

Clinical research and clinical trials
Project
using the iPS cell stock

CiRA Foundation
Activity Report

Clinical research and clinical trials using the CiRA̲F iPS cell stock are being conducted for various diseases and disorders. A list of
diseases and the cell types made from the iPS cell stock used to study them are given below. All iPS cells are reprogrammed blood cells.

Past provisions of the iPS Cell StockDiﬀerentiated cells
Neurons

Parkinson s Disease

Retinal cells

Macular degeneration
Retinitis Pigmentosa

Patients
anddonorsiPS Cells（collecting blood）

Red … On goingBlue … Approved
Green … Planned*Clinical research and clinical trials are conductedby medical institutions to which CiRA̲F provides
the stock and not by CiRA̲F itself.

FutureCorneal cells

Corneal epithelial
stem cell deﬁciency

Heart Muscle cells

Ischemic cardiomyopathy
Severe heart failure

Cartilage

Articular cartilage damage

Neural stem cells

Spinal cord injury

Platelets

Thrombocytopenia

Immune cells

Hematologic cancer,
Solid carcinoma

Hepatocytes

Liver failure

Pancreatic β-cells

Type 1 Diabetes

Kidney cells

Renal failure*As of June 30, 2021. Totals include
projects when the iPS cell stock was
operated by the Center for iPS Researchand Application (CiRA), Kyoto University.

HLA-homozygous
iPS cell stock for research
50 projects, 58 institutions

HLA-genome-edited
iPS cell stock for research
14 projects, 13 institutions

HLA-homozygous
iPS cell stock for clinical use
27 projects, 28 institutions
We have or are scheduled to provide
iPS cell stock to the organizations below.

CHA University（South Korea）
Novo Nordisk（Denmark）

The Present and Future of iPS Cells

Below are current projects at CiRA̲F.

HLA-homozygous
iPS cell stock（currently covers 40% ofJapanese population）

Donors（collecting blood）

iPS cells
Diﬀerentiatedcells

Patients

HLA-genome-edited
iPS cell stock（aims to cover most of
the world population）

Donors（collecting blood）

iPS cells＋
Genome editingDiﬀerentiatedcells

my iPS Project（personalized iPS cells）

Donors（collecting blood）

iPS cells
Diﬀerentiatedcells

Patients

Immune rejection is a common problem in any type oftransplantation. This rejection is the result of HLA (humanleukocyte antigen) mismatching. To minimize the risk ofmismatching and increase the number of eligible patients, CiRA̲F
is developing an HLA-homozygous iPS cell stock.

Since there are tens of thousands of possible HLA antigen
combinations, it is very costly and time consuming to recruit
enough HLA-homozygous donors (see above) to prepare iPS cells
suitable for transplantation therapies to all patients with minimalrisk of immune rejection. Therefore, CiRA̲F is genome editing iPS
cells. Those iPS cells are currently for research use only, but we
expect to obtain approval for medical use in 2022.

iPS cells are made by reprogramming the patient's own cells.
The manufacturing of my iPS cells is planned to start in 2025.
Before that, we need to clear many technical issues, such as
automating the cell culture and reducing the cost.
Before treatment, the iPS cells and the diﬀerentiated cells must be
thoroughly tested to assure that they are safe.
CiRA̲F has decided to set up a base facility for this project at the"International Hub for Healthcare Innovation" in Osaka.

*When actually used to treat patients,
iPS cells must be diﬀerentiated beforehand.

CiRA Foundation
Activity Report vol.2

CiRA Foundation

Published on July 21, 2021

www.cira-foundation.or.jp

53 Shogoin kawahara-cho,
Sakyo-ku, Kyoto 606-8397, Japan.

Message from
Representative
Director of CiRA̲F

vol.

2

July 2021

Bridging laboratory results to clinical results
In April 2020, we began the CiRA Foundation, and weare now celebrating our ﬁrst anniversary. I would like toexpress my heartfelt gratitude to all of you for yourcontinued support.
Fourteen years have passed since the announcement of
human iPS cells. The Center for iPS Cell Research andApplication (CiRA), Kyoto University, has been acting as a"bridge" to transfer technologies for iPS cell production
and quality evaluation to industry. However, because
CiRA is part of a university and therefore founded as an
educational and research institution, its activities are
restricted.
The Foundation has taken over the responsibility of the"bridge" from CiRA and continues working to achieve its
mission of providing top iPS cell technologies at aﬀordable
prices. To assist research institutions and companies, we
provide iPS cells for regenerative medicine free of charge
to non-proﬁt institutions and at a low price to for-proﬁt
institutions. Furthermore, we will contribute to therealization of regenerative medicine by developing

Anniversary

automated cell manufacturing technologies and by sharing
versatile technologies and information with companies.
We are just starting our journey on the road to thepractical application of iPS cells. Thanks to the eﬀorts ofmany researchers, clinical trials have begun to verify thesafety and eﬃcacy of several iPS cell products. Our work
will help commence even more clinical trials. We lookforward to your continued support and cooperation.

Shinya YAMANAKA
Representative Director

CiRA̲F s 1st Anniversary

https://www.youtube.com/
watch?v=7yUPGlsBS5M&t=236s

On April 23, 2021, we held an online session to recognize our anniversary and to express
our gratitude. Thank you very much to everyone who participated in the event.

Lecture content①The signiﬁcance of the CiRA Foundation and Future of iPS cells
Speaker：Shinya YAMANAKA, Representative Director②Why we need your support
Speaker：Fumitaka WATANABE, Philanthropy Oﬃce Leader

On the day, we also welcomed questions and comments from participants.
Due to time constraints, we were not able to answer all of the questions, but we had a great
opportunity to understand people s interests in our work and on iPS cells in general.
It was also a great surprise to receive questionnaire responses from more than 80% of participants after the session. Many included
messages of support to the Foundation. These messages have been shared with our staﬀ members, and we thank all those who wrotethem.
We will continue to regularly send out information about our work in accordance with our mission, Walking with patients to leadsociety .

News

Activities of CiRA̲F

January 2021

Obtained a manufacturing license based on the PMD Act

Principles

The Four Guiding Principles of the Foundation

In January 2021, CiRA̲F announced four guidelines.
These guidelines serve our work every day.

CiRA̲F has obtained a manufacturing license (general category) for regenerative medicine products
based on the Pharmaceutical Aﬀairs and Medical Devices Act (PMD Act) in Japan. By obtaining thislicense, we can expand the range of collaborations with companies and contribute to theregenerative medicine industry.

February 2021

Basic agreement with Thyas for the manufacturing of
iPS cell-derived T cells
CiRA̲F has signed a basic agreement for the manufacturing of autologous regenerated T cells by
Thyas. Joint research with Thyas for the my iPS Project to produce autologous regenerated T cells
has also started.

March 2021

PHILOSOPHY

VALUESWalking withpatients tolead society

Top iPS cell
technologies
at aﬀordable prices

People are
our driving force

To advance,
to appreciate,
to shareWe understand that to be a

To bring iPS cells to the clinic

Why do we do this work?

We believe the goals of ourleader in medical technology,

as quickly as possible, we will

Because of people. These

employees and our partners are

we must be always developing

bring technologies for cell

people include, of course,

the same: to provide high quality

our technology. But our mostmanufacturing and quality

patients, but their families too.

iPS cells at aﬀordable prices.

important motivation is thecontrol to industry.

They also include our partners

Because we share the same

patient.

As technology develops, so will

who hold our passion andpurpose, we recognize theThus, to us, development is not

we.

ideals.

importance of advancing eachsimply pushing technology

By collecting and sharing

If it were only us, it would beother s skills, appreciating eachforward. It is standing withinformation on research andeasy to give up. But because

other s roles, and sharing eachApplication (CiRA), Kyoto University. CiRA̲F is responsible for the manufacturing, the non-clinicalpatients and understanding

development, we will raise thepeople are our driving force, weother s accomplishments.

safety evaluation, and the quality assessment of human iPS cell-derived platelets.

their needs. This is our mission.

quality, lower the cost, anddevote ourselves to raising theWith priority to the patients, we

ultimately advance clinicalquality of iPS cells at aﬀordable

will take on our challenges.

applications.

prices and acting as a bridge to

Began accepting applications for the free provision of
iPS cells made from convalescent COVID-19 patients
The collaboration team to the left has established iPS cells from the peripheral blood of patients who
have recovered from SARS-CoV-2 infection. These iPS cell lines are being distributed free of charge
to companies and research institutes for the study of COVID-19, such as the onset mechanism.

April 2021

Platelets derived from the CiRA Foundation iPS Cell
Stock enter an industry-led clinical trial
Evaluation of the clinical trial plan has been completed. This project is in collaboration with
Megakaryon Corporation, Kyoto University Hospital, and the Center for iPS Cell Research and

May 2021

CODE ofCONDUCTMISSION

industry.

First shipment of our iPS cell stock to an overseas partner

TopicsMilestones to date

Our iPS cell stock arrived at CHA University, South Korea, safely at the end of May this year. We alsoplan to provide HLA-homozygous iPS cell stock to Novo Nordisk, which is a global pharmaceuticalcompany headquartered in Denmark.

In order to handle a

April 1, 2020.

large amount of

CiRA̲F started its activities with 83

important researchpeople reassigned from CiRA and 5

July 2021

A base facility for the my iPS Project has been selected
project, please see "The Present and Future of iPS Cells" on page 4. The facility will be open to thefor this day.

who aspire to become researchers in the future.

As a separate entity from Kyoto
University, we had to establish new
employment rules, launch our
website, and make other changes, allof which required a concerted eﬀort
by our staﬀ.

*In addition to the above news, we are working with many companies and organizations on donations.
Thank you very much for your cooperation.

a network system

infection, we had to cancel theWe have decided to locate the base facility for the my iPS Project in Osaka. For more details on thewhere people can learn about iPS cells, but also a place to disseminate information to young people

data, the creation of

Due to the spread of COVID-19
unveiling ceremony that we preparedpublic, donors, and visitors from research institutes, etc. We hope that it will not only be a placeand manufacturing

new hires.

was also critical at
the time of inception.
We received support from a number ofcompanies to prepare the system so that
we could start operating about 100
computer terminals within a preparation
time of ﬁve months, including theconceptualization.

Our growth and activities arebecause of the cooperationof many people every day.
We hope that you willcontinue to support us in oureﬀorts.

News

Activities of CiRA̲F

January 2021

Obtained a manufacturing license based on the PMD Act

Principles

The Four Guiding Principles of the Foundation

In January 2021, CiRA̲F announced four guidelines.
These guidelines serve our work every day.

CiRA̲F has obtained a manufacturing license (general category) for regenerative medicine products
based on the Pharmaceutical Aﬀairs and Medical Devices Act (PMD Act) in Japan. By obtaining thislicense, we can expand the range of collaborations with companies and contribute to theregenerative medicine industry.

February 2021

Basic agreement with Thyas for the manufacturing of
iPS cell-derived T cells
CiRA̲F has signed a basic agreement for the manufacturing of autologous regenerated T cells by
Thyas. Joint research with Thyas for the my iPS Project to produce autologous regenerated T cells
has also started.

March 2021

PHILOSOPHY

VALUESWalking withpatients tolead society

Top iPS cell
technologies
at aﬀordable prices

People are
our driving force

To advance,
to appreciate,
to shareWe understand that to be a

To bring iPS cells to the clinic

Why do we do this work?

We believe the goals of ourleader in medical technology,

as quickly as possible, we will

Because of people. These

employees and our partners are

we must be always developing

bring technologies for cell

people include, of course,

the same: to provide high quality

our technology. But our mostmanufacturing and quality

patients, but their families too.

iPS cells at aﬀordable prices.

important motivation is thecontrol to industry.

They also include our partners

Because we share the same

patient.

As technology develops, so will

who hold our passion andpurpose, we recognize theThus, to us, development is not

we.

ideals.

importance of advancing eachsimply pushing technology

By collecting and sharing

If it were only us, it would beother s skills, appreciating eachforward. It is standing withinformation on research andeasy to give up. But because

other s roles, and sharing eachApplication (CiRA), Kyoto University. CiRA̲F is responsible for the manufacturing, the non-clinicalpatients and understanding

development, we will raise thepeople are our driving force, weother s accomplishments.

safety evaluation, and the quality assessment of human iPS cell-derived platelets.

their needs. This is our mission.

quality, lower the cost, anddevote ourselves to raising theWith priority to the patients, we

ultimately advance clinicalquality of iPS cells at aﬀordable

will take on our challenges.

applications.

prices and acting as a bridge to

Began accepting applications for the free provision of
iPS cells made from convalescent COVID-19 patients
The collaboration team to the left has established iPS cells from the peripheral blood of patients who
have recovered from SARS-CoV-2 infection. These iPS cell lines are being distributed free of charge
to companies and research institutes for the study of COVID-19, such as the onset mechanism.

April 2021

Platelets derived from the CiRA Foundation iPS Cell
Stock enter an industry-led clinical trial
Evaluation of the clinical trial plan has been completed. This project is in collaboration with
Megakaryon Corporation, Kyoto University Hospital, and the Center for iPS Cell Research and

May 2021

CODE ofCONDUCTMISSION

industry.

First shipment of our iPS cell stock to an overseas partner

TopicsMilestones to date

Our iPS cell stock arrived at CHA University, South Korea, safely at the end of May this year. We alsoplan to provide HLA-homozygous iPS cell stock to Novo Nordisk, which is a global pharmaceuticalcompany headquartered in Denmark.

In order to handle a

April 1, 2020.

large amount of

CiRA̲F started its activities with 83

important researchpeople reassigned from CiRA and 5

July 2021

A base facility for the my iPS Project has been selected
project, please see "The Present and Future of iPS Cells" on page 4. The facility will be open to thefor this day.

who aspire to become researchers in the future.

As a separate entity from Kyoto
University, we had to establish new
employment rules, launch our
website, and make other changes, allof which required a concerted eﬀort
by our staﬀ.

*In addition to the above news, we are working with many companies and organizations on donations.
Thank you very much for your cooperation.

a network system

infection, we had to cancel theWe have decided to locate the base facility for the my iPS Project in Osaka. For more details on thewhere people can learn about iPS cells, but also a place to disseminate information to young people

data, the creation of

Due to the spread of COVID-19
unveiling ceremony that we preparedpublic, donors, and visitors from research institutes, etc. We hope that it will not only be a placeand manufacturing

new hires.

was also critical at
the time of inception.
We received support from a number ofcompanies to prepare the system so that
we could start operating about 100
computer terminals within a preparation
time of ﬁve months, including theconceptualization.

Our growth and activities arebecause of the cooperationof many people every day.
We hope that you willcontinue to support us in oureﬀorts.

CiRA Foundation

Clinical research and clinical trials
Project
using the iPS cell stock

CiRA Foundation
Activity Report

Clinical research and clinical trials using the CiRA̲F iPS cell stock are being conducted for various diseases and disorders. A list of
diseases and the cell types made from the iPS cell stock used to study them are given below. All iPS cells are reprogrammed blood cells.

Past provisions of the iPS Cell StockDiﬀerentiated cells
Neurons

Parkinson s Disease

Retinal cells

Macular degeneration
Retinitis Pigmentosa

Patients
anddonorsiPS Cells（collecting blood）

Red … On goingBlue … Approved
Green … Planned*Clinical research and clinical trials are conductedby medical institutions to which CiRA̲F provides
the stock and not by CiRA̲F itself.

FutureCorneal cells

Corneal epithelial
stem cell deﬁciency

Heart Muscle cells

Ischemic cardiomyopathy
Severe heart failure

Cartilage

Articular cartilage damage

Neural stem cells

Spinal cord injury

Platelets

Thrombocytopenia

Immune cells

Hematologic cancer,
Solid carcinoma

Hepatocytes

Liver failure

Pancreatic β-cells

Type 1 Diabetes

Kidney cells

Renal failure*As of June 30, 2021. Totals include
projects when the iPS cell stock was
operated by the Center for iPS Researchand Application (CiRA), Kyoto University.

HLA-homozygous
iPS cell stock for research
50 projects, 58 institutions

HLA-genome-edited
iPS cell stock for research
14 projects, 13 institutions

HLA-homozygous
iPS cell stock for clinical use
27 projects, 28 institutions
We have or are scheduled to provide
iPS cell stock to the organizations below.

CHA University（South Korea）
Novo Nordisk（Denmark）

The Present and Future of iPS Cells

Below are current projects at CiRA̲F.

HLA-homozygous
iPS cell stock（currently covers 40% ofJapanese population）

Donors（collecting blood）

iPS cells
Diﬀerentiatedcells

Patients

HLA-genome-edited
iPS cell stock（aims to cover most of
the world population）

Donors（collecting blood）

iPS cells＋
Genome editingDiﬀerentiatedcells

my iPS Project（personalized iPS cells）

Donors（collecting blood）

iPS cells
Diﬀerentiatedcells

Patients

Immune rejection is a common problem in any type oftransplantation. This rejection is the result of HLA (humanleukocyte antigen) mismatching. To minimize the risk ofmismatching and increase the number of eligible patients, CiRA̲F
is developing an HLA-homozygous iPS cell stock.

Since there are tens of thousands of possible HLA antigen
combinations, it is very costly and time consuming to recruit
enough HLA-homozygous donors (see above) to prepare iPS cells
suitable for transplantation therapies to all patients with minimalrisk of immune rejection. Therefore, CiRA̲F is genome editing iPS
cells. Those iPS cells are currently for research use only, but we
expect to obtain approval for medical use in 2022.

iPS cells are made by reprogramming the patient's own cells.
The manufacturing of my iPS cells is planned to start in 2025.
Before that, we need to clear many technical issues, such as
automating the cell culture and reducing the cost.
Before treatment, the iPS cells and the diﬀerentiated cells must be
thoroughly tested to assure that they are safe.
CiRA̲F has decided to set up a base facility for this project at the"International Hub for Healthcare Innovation" in Osaka.

*When actually used to treat patients,
iPS cells must be diﬀerentiated beforehand.

CiRA Foundation
Activity Report vol.2

CiRA Foundation

Published on July 21, 2021

www.cira-foundation.or.jp

53 Shogoin kawahara-cho,
Sakyo-ku, Kyoto 606-8397, Japan.

Message from
Representative
Director of CiRA̲F

vol.

2

July 2021

Bridging laboratory results to clinical results
In April 2020, we began the CiRA Foundation, and weare now celebrating our ﬁrst anniversary. I would like toexpress my heartfelt gratitude to all of you for yourcontinued support.
Fourteen years have passed since the announcement of
human iPS cells. The Center for iPS Cell Research andApplication (CiRA), Kyoto University, has been acting as a"bridge" to transfer technologies for iPS cell production
and quality evaluation to industry. However, because
CiRA is part of a university and therefore founded as an
educational and research institution, its activities are
restricted.
The Foundation has taken over the responsibility of the"bridge" from CiRA and continues working to achieve its
mission of providing top iPS cell technologies at aﬀordable
prices. To assist research institutions and companies, we
provide iPS cells for regenerative medicine free of charge
to non-proﬁt institutions and at a low price to for-proﬁt
institutions. Furthermore, we will contribute to therealization of regenerative medicine by developing

Anniversary

automated cell manufacturing technologies and by sharing
versatile technologies and information with companies.
We are just starting our journey on the road to thepractical application of iPS cells. Thanks to the eﬀorts ofmany researchers, clinical trials have begun to verify thesafety and eﬃcacy of several iPS cell products. Our work
will help commence even more clinical trials. We lookforward to your continued support and cooperation.

Shinya YAMANAKA
Representative Director

CiRA̲F s 1st Anniversary

https://www.youtube.com/
watch?v=7yUPGlsBS5M&t=236s

On April 23, 2021, we held an online session to recognize our anniversary and to express
our gratitude. Thank you very much to everyone who participated in the event.

Lecture content①The signiﬁcance of the CiRA Foundation and Future of iPS cells
Speaker：Shinya YAMANAKA, Representative Director②Why we need your support
Speaker：Fumitaka WATANABE, Philanthropy Oﬃce Leader

On the day, we also welcomed questions and comments from participants.
Due to time constraints, we were not able to answer all of the questions, but we had a great
opportunity to understand people s interests in our work and on iPS cells in general.
It was also a great surprise to receive questionnaire responses from more than 80% of participants after the session. Many included
messages of support to the Foundation. These messages have been shared with our staﬀ members, and we thank all those who wrotethem.
We will continue to regularly send out information about our work in accordance with our mission, Walking with patients to leadsociety .