[Updated] JCR Pharmaceuticals presents preclinical gene therapy data at ASGCT 28th Annual Meeting
Hyogo, Japan – May 15, 2025 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy research programs at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, being held May 13-17, 2025, in New Orleans, LA. In an oral presentation, the JCR researcher reported that the Company’s proprietary J-Brain Cargo® (JBC) technology enables the efficient delivery of an adeno-associated virus (AAV) gene therapy across the bloodbrain barrier (BBB) and into the central nervous system (CNS) in mice, monkeys, and several animal models of CNS diseases.
We have successfully developed JUST-AAV, a novel AAV vector platform technology designed to enhance targeted delivery and reduce liver tropism, thereby improving safety and efficacy of AAVbased gene delivery technologies to the CNS. JUST-AAV encompasses a range of vector types optimized for various target tissues, including liver-sparing, muscle-targeting, and brain-targeting variants. This proprietary technology holds significant promise for advancing the field of AAVbased gene therapy.
“These findings represent a significant advancement toward new treatments for previously challenging CNS diseases,” said Hiroyuki Sonoda, Ph.D., Director, Senior Managing Executive Officer, and Executive Director, Research Division at JCR Pharmaceuticals. “Our preclinical data demonstrate that our brain-targeting JUST-AAV technology delivers therapeutic agents to the central nervous system far more efficiently than conventional AAV9, while significantly reducing liver accumulation. This enhanced safety profile greatly increases the potential for clinical translation. This research is a crucial step in our ongoing commitment to developing innovative solutions for unmet medical needs.”
JCR showcased the following presentation:
Incorporation of transferrin receptor binder and surface mutations into AAV enables efficient brain delivery and reduced liver tropism
Presenter: Yuhei Ashida (JCR Pharmaceuticals)
Researchers successfully created a brain-targeting AAV vector (brain-targeting JUST-AAV) by incorporating a miniaturized antibody that binds to the transferrin receptor, into the AAV capsid. Furthermore, unique modifications to the AAV capsid sequence significantly reduced AAV vector accumulation in the liver, a known source of adverse effects. In mouse studies, the JUST-AAV vector achieved 77-fold higher expression of green fluorescent protein (GFP) in the brain compared to AAV9, while reducing the tropism to the liver by 99%. To further enhance brain targeting and BBB permeability, the researchers incorporated additional molecules that bind to receptors other than the transferrin receptor into the AAV capsid. In monkey studies, this bispecific vector demonstrated an improved gene delivery efficiency to the brain by several orders of magnitude compared with AAV9, while reducing infection in potentially problematic tissues such as the liver and dorsal root ganglia by more than 90%.
Application of JUST-AAV to a mouse model of neuronal ceroid lipofuscinosis resulted in the disappearance of symptoms such as seizures and prolonged lifespan to an extent of a functional cure. These results suggest that the newly developed JUST-AAV technology offers the potential for safer and more efficient gene therapy than conventional gene delivery vectors.
About the American Society of Gene and Cell Therapy (ASGCT)
The American Society of Gene and Cell Therapy (ASGCT) is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates, and other professionals. The mission of the ASGCT is to advance knowledge, awareness, and education, leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. For more information, please visit www.asgct.org.
About the J-Brain Cargo® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder.
JUST-AAV
JUST-AAV is a proprietary platform technology that utilizes modified adeno-associated virus (AAV) vectors. The technology entails insertion of miniaturized antibodies against receptors on selected tissues, organs or the blood-brain barrier onto the capsid surface, enhancing targeted delivery to those tissues and organs. Further capsid modifications minimize off-target effects and improve safety. The name is derived from “JCR” “Ultimate destination of organ” “Safeguarding against off-target delivery” and “Transformative technology” reflecting its potential for broad application across various diseases.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/index.html.
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Corporate Communications
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JCR Pharmaceuticals announces chairman and president transition as of April 1, 2026
Hyogo, Japan – May 13, 2025 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) today announced that, effective April 1, 2026, the company will implement a new leadership structure. The changes were approved at the Board of Directors meeting held earlier today. Under the new structure, Toru Ashida will assume the role of Chairman, and Hiroyuki Sonoda will become President. Current Chairman, President and CEO, Shin Ashida, will transition to the role of Founder and Director.
Changes in Representative Directors (including Chairman and President)
1. New Assignments of Representative Directors
| Name | New position | Current position |
|---|---|---|
| Toru Ashida | Representative Director Chairman |
Director, Senior Managing Executive Officer, Sales, Executive Director, Sales Division |
| Hiroyuki Sonoda | Representative Director President |
Director, Senior Managing Executive Officer, Research, Executive Director, Research Division |
| Shin Ashida | Director, Founder | Representative Director Chairman, President and CEO |
2. Reason for Changes
Since founding JCR in September 1975, Shin Ashida has played a pivotal role in shaping the company’s growth, grounded in a deep and enduring commitment to innovation in biopharmaceutical research and manufacturing. As the company marks its 50th anniversary this September, this leadership transition reflects a generational shift aimed at further strengthening the management structure and driving long-term corporate value.
Details regarding other executive appointments will be announced once internal discussions have been concluded.
3. Effective Date
April 1, 2026
(Reference)
Brief Personal History of New Chairman
Name: Toru Ashida
Birthdate: October 31, 1968 (56 years old)
Birthplace: Ashiya City, Hyogo Prefecture, Japan
Education: March 1992 Graduated from Keio University, Faculty of Business and Commerce
Career
April 1992 - Joined Nippon Life Insurance Company
April 2002 - Appointed Representative Director and President at the establishment of JBS Co., Ltd. (clinical trial site support organization)
January 2014 - Joined the Company
July 2014 - Appointed Corporate Officer of the Company Executive Director of Corporate Business Support Division and Director of Corporate Strategy Department of the Company
April 2016 - Head of Office of the President of the Company
June 2018 - Appointed Senior Executive Director of the Company Head of Quality Assurance Division, Corporate Planning Division, and Medical Affairs Department of the Company
June 2019 - In charge of Corporate Strategy Head of Quality Assurance Division, Corporate Planning Division, Administration Division, and Medical Affairs Department of the Company
April 2020 - Executive Director of Sales Division of the Company (to present)
June 2020 - Appointed Vice President of the Company and in charge of Sales Division
June 2021 - Appointed Senior Vice President of the Company and in charge of Sales and Administration
April 2023 - In charge of Sales (to present)
June 2024 - Appointed Director and Senior Managing Executive Officer of the Company (to present)
Number of Shares Held
360,308 shares
Brief Personal History of New President
Name: Hiroyuki Sonoda, Ph.D.
Birthdate: June 15, 1978 (46 years old)
Birthplace: Maizuru City, Kyoto Prefecture, Japan
Education: September 2010 Graduated from Department of Chemical Science and Engineering Faculty of Engineering Graduate School of Engineering Kobe University (Doctor of Engineering)
Career
April 2003 - Joined the Company
April 2016 - Director of Corporate Planning Division (In charge of Research) of the Company
October 2017 - Leader of Frontier Research Unit and Director of Corporate Planning Division (In charge of Research) of the Company
April 2018 - Executive Director of Research Planning Division of the Company
June 2018 - Appointed Corporate Officer of the Company
June 2020 - Appointed Senior Executive Director of the Company and in charge of Research and Development Division Executive Director of Research Division, and Director of Drug Discovery Research Institute
June 2021 Appointed Vice President of the Company and in charge of Research and Corporate Strategy Executive Director of Research Division (to present)
October 2022 Representative Director and President of AlliedCel Corporation (Joint venture with Sysmex Corporation) (to present)
April 2023 In charge of Research (to present)
June 2024 Appointed Director and Senior Managing Executive Officer of the Company (to present)
Number of Shares Held
68,320 shares
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our50-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, HurlerScheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/.
Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com
JCR Pharmaceuticals announces new corporate philosophy and core values
Hyogo, Japan – May 13, 2025 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) has renewed its corporate philosophy and core values to mark the 50th anniversary of its founding and to set a clearer direction for the future.
JCR has long focused on rare and genetic diseases, earning recognition as a specialty pharmaceutical company built on scientific innovation. Through our work with protein, cell-based, and gene technologies, we’ve developed therapies and platforms that are expanding the possibilities of treatment. At the heart of it all is a simple promise—to improve people’s health through the medicines we create.
As we mark this milestone, we’ve taken a moment to reflect on what truly matters—our role in society, what we stand for, and how we move forward. This reflection has led us to renew our corporate philosophy, bringing even more clarity to our purpose and the values we share.
Our Corporate Philosophy
"We create treatments that go beyond rare disease to solve the world's most complex healthcare challenges”
This renewed philosophy is built on two ideas we care deeply about - continuing to explore areas of medicine where no treatments yet exist, and expanding the reach of our innovations so they can support more patients, wherever they are.
JCR has long focused on these three values, and they have shaped the way we think and act— Reliability, Confidence, and Belief. As we move forward, we’ve taken a step back to reflect on what they mean today and how they will help guide us well into the future.
Our Core Values
1. Putting people first
Our community is our priority. Patients, families, physicians and our employees are at the heart of what we do.
2. Forging our own path
Unshackled from convention, we create new solutions that no-one else can.
3. Always advancing
We don’t stand still. Our research continuously pushes boundaries to deliver for patients and families.
4. Committed to excellence
We deliver the highest standards for partners, patients and employees.
With a renewed sense of purpose, each of us at JCR is moving forward—thinking boldly, acting with intention, and striving to close the gap in unmet medical needs. This is how we grow— together.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, HurlerScheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level.
Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com