Moises Zanetti

JCR Pharmaceuticals marks 50 years of innovation with launch of one-of-a-kind global website

Hyogo, Japan – June 6, 2025 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the launch of its new global website. As the company turns 50, it is marking its growing footprint in Japan, the U.S., Europe, and Latin America with a unique website tailored for global audiences that reflects its passion for both science and humanity and brings the JCR brand to life for patients, physicians, and partners worldwide.

“At JCR, we are committed to advancing science and delivering on the treatments for patients with unmet needs around the world,” said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. “In September, we celebrate the 50th anniversary of the company I founded in 1975. The launch of our new global website reflects our evolution from an innovative startup in Japan to a global biopharmaceutical company. We remain dedicated to developing therapies for people with rare and genetic diseases and partnering with those who share our vision of transforming care for patients and families living with once untreatable diseases.”

The site features streamlined user interface and curated content for patients, physicians, partners, and investors – reinforcing JCR’s commitment to bridge the gap between complex science and unmet needs of patients and families.

For the past 50 years, JCR has built a reputation for the development of innovative new therapeutic approaches, including its more recent J-Brain Cargo® technology, a proprietary bloodbrain barrier-penetrating technology that can deliver biotherapeutics into the central nervous system (CNS). This platform underpins several investigational pre-clinical and clinical staged therapies in JCR’s pipeline, beginning with lysosomal storage disorders (LSDs), and it reflects the company’s ongoing commitment to advancing treatments for CNS-related diseases across neurodegeneration, neuro-inflammation, and neuro-oncology conditions. JCR continues to strengthen its global research, development, and manufacturing capabilities, with a focus on addressing unmet medical needs and expanding therapeutic possibilities.

Learn more about JCR, its technologies, and clinical development programs on its global website: https://jcrpharm.com/.

About the J-Brain Cargo® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, JBrain Cargo®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for the treatment a lysosomal storage disorder is IZCARGO® (INN: pabinafusp alfa).

About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world’s most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., 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. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website: https://jcrpharm.com/.

Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com

by Moises Zanetti

[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.

Cautionary Statement Regarding Forward-Looking Statements
This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions.

This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue.

Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.

Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com

by Moises Zanetti

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

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

by Moises Zanetti