JCR Pharmaceuticals to Present at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting
Hyogo, Japan – May 11, 2026 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced today that it will present preclinical data from its novel platform technologies, including JUST-AAV gene therapy, in oral and poster sessions at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting, being held May 11-15, 2026, in Boston, Massachusetts. Additionally, Alexion, AstraZeneca Rare Disease, will present preclinical data from a research collaboration that applies the JUST-AAV technology in an oral session.
The details of the presentations are listed below, and the full program can be found on the ASGCT congress website at https://annualmeeting.asgct.org/.
Title: Development of an AAV-Based Gene Therapy for GM1 Gangliosidosis Using a Transferrin Receptor Antibody–Fused Enzyme with Markedly Enhanced Therapeutic Efficacy
Session: Neurologic Diseases VI
Presentation Number: 391
Date/Time: Thursday, May 14, 4:45 PM – 5:00 PM ET
Presenter: Saki Matsushima, Ph.D. (The Jikei University School of Medicine, Division of Gene Therapy, Research Center for Medical Sciences, Tokyo, Japan)
Title: Long-Term Efficacy and Neuroprotection by Systemically Administered, CNS-Targeting AAV Capsids in Mouse Models of Neuronal Ceroid Lipofuscinosis (CLN1 and CLN2)
Session: Poster Reception
Poster Number: 3460
Date/Time: Thursday, May 14, 5:00 PM – 6:30 PM ET
Lead Author: Tomoki Hirashima (JCR Pharmaceuticals)
Title: AAV Capsid Presenting a Miniaturized Anti–Transferrin Receptor Antibody Enables Broad CNS, Liver-detargeted Biodistribution
Session: AAV Capsid Engineering IV
Presentation Number: 427
Date/Time: Friday, May 15, 9:15 AM – 9:30 AM ET
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 JUST-AAV Platform Technology
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 liver tropism and potentially mitigate hepatoxicity, which is a commonly observed safety concern. 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 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 partners, patients and employees. We strive 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
JCR Pharmaceuticals initiates phase III dose comparison study of GROWJECT™ S.C. injection 12mg in Japan
Hyogo, Japan – April 13, 2026 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced that the first participant has been dosed in a Phase III dose comparison study of GROWJECT™ S.C. injection 12mg (somatropin, a recombinant therapy) in pediatric patients with growth hormone deficiency short stature in Japan.
In Japan, standard treatment for pediatric growth hormone deficiency short stature includes daily subcutaneous administration of recombinant human growth hormone (HGH) at 0.175 mg/kg/week, as well as once-weekly long-acting recombinant HGH products. Outside of Japan, dose adjustment according to patient response is permitted for daily HGH products, and in the United States, pediatric patients with growth hormone deficiency short stature may receive adjusted doses (0.175–0.3 mg/kg/week).1
To help improve final adult height and quality of life (QoL) in Japan by narrowing the gap with international dosing practices, JCR has initiated development of a dosing regimen that allows dose adjustment in Japan.
This study will compare the efficacy and safety of continuous administration of the study drug at 0.3 mg/kg/week versus 0.175 mg/kg/week in pediatric patients with growth hormone deficiency short stature, while also evaluating the superiority of the higher-dose regimen. Details of the study are available through the Japan Registry of Clinical Trials (jRCT): (JR-401G-301 Study: jRCT2051250213).
“I am pleased to see progress in therapies for children,” said Dr. Kenichi Kashimada, Head, Division of Endocrinology and Metabolism, National Center for Child Health and Development. “Through this study, treatment with daily HGH products will be reexamined against international dosing standards rather than the conventional standard dose used in Japan. I hope this will lead to new treatment strategies and help improve final adult height and QoL for pediatric patients with growth hormone deficiency short stature. Going forward, safe and sustainable treatment approaches tailored to each patient are needed.”
JCR remains committed to contributing to the field of growth hormone therapy by maintaining a stable supply of high-quality pharmaceuticals and providing a broad range of treatment options for growth disorders to better meet the needs of patients.
About GROWJECT™
GROWJECT™ is an injectable preparation containing recombinant human growth hormone, first launched in Japan by JCR in 1995. The dosage is administered subcutaneously six to seven times per week, adjusted according to the indicated condition.
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 partners, patients and employees. We strive to expand the possibilities for patients while accelerating medical advancement at a global level.
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.
Reference
1. Tanaka T. Changes, limitations, and prospects of adult height in GH treatment for Japanese GHD patients. Clin Pediatr Endocrinol. 2022;31(4):211-24.
Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com
JCR Pharmaceuticals launches redesigned corporate website
Hyogo, Japan – April 1, 2026 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the redesign of its corporate website.
The website features a fully revised structure and visual design intended to make JCR’s business activities and corporate perspective easier for stakeholders to understand. It has also been optimized for a wide range of devices, including smartphones and tablets. Building on the visual identity introduced with the launch of JCR’s global website in June 2025, both the Japanese and English versions of the Japan website have been refreshed. The design also reflects JCR’s corporate slogan, “Life is Rare,” to communicate its values and direction consistently to stakeholders in Japan and around the world.
Key features of the redesigned website:
A clearer view of JCR’s business
Reorganizing the site to make the role, features, and strengths of each business easier to understand.
Clearer communication of JCR’s message
Presenting JCR’s corporate philosophy, business approach, direction, and value more clearly.
Easier access to information
Redesigning navigation to help visitors access the information they need more smoothly.
Optimized for a better viewing experience
Providing comfortable viewing across devices, including smartphones and tablets.
JCR remains committed to appropriate disclosure and to further enhancing its corporate value.
Website Links:
Japanese website: https://www.jcrpharm.co.jp/
English website: https://www.jcrpharm.co.jp/en/
Global website: https://www.jcrpharm.com/
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 partners, patients and employees. We strive 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
JCR establishes Advanced Biopharmaceutical Research Institute
Hyogo, Japan – February 26, 2026 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, will establish the Advanced Biopharmaceutical Research Institute at Creative Lab Kobe (CLIK) within the Kobe Biomedical Innovation Cluster (KBIC) to advance biopharmaceutical research and core technologies.
The institute will focus on addressing unmet medical needs, including neurodegenerative and rare diseases. In addition to advancing existing research and development (R&D) initiatives, it will pursue novel therapeutic strategies and innovative drug delivery technologies.
Based in the KBIC, which brings together industry, academia, and government, the institute will strengthen collaboration with academic institutions and external research organizations while developing new core technologies for future biopharmaceutical research.
Overview of the Advanced Biopharmaceutical Research Institute
Location: Creative Lab Kobe (CLIK), Rooms 601–602, 6-3-7 Minatojima-Minamimachi, Chuo-ku, Kobe, Japan
Planned Opening: April 1, 2026
Director: Kenichi Takahashi, Ph.D., Scientific Expert Fellow
About the Kobe Biomedical Innovation Cluster
Launched by the City of Kobe in 1998 as part of post Great Hanshin Awaji Earthquake reconstruction efforts, the KBIC has grown over nearly 30 years into one of Japan’s largest biomedical cluster. The area brings together academic institutions, medical companies, RIKEN research centers, advanced hospitals, and research infrastructure including the supercomputer Fugaku, creating a strong environment for biomedical research and development.
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 partners, patients and employees. We strive 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
JCR Pharmaceuticals supports Rare Disease Day 2026
February 28, 2026, marks Rare Disease Day (RDD), the world’s largest global awareness campaign dedicated to improving understanding of rare diseases and advancing diagnosis, treatment, and quality of life for those affected and their families. Recognizing the importance of this global effort, JCR will once again support RDD 2026 in Japan.
JCR has supported RDD Japan since 2015 and will again participate through awareness and engagement initiatives.
Featured Session at RDD Tokyo event
JCR will present a featured session at the RDD Tokyo event on February 28 (17:30-18:00, scheduled) at KABUTO ONE 4F Hall and Conference floor, 7-1 Nihonbashi Kabutocho, Chuo-ku, Tokyo, Japan.
The session will include a video presentation titled “Sport Connects. Bringing Hope to Rare Diseases” featuring professional tennis players, Masamichi Imamura and Shinji Hazawa, official ambassadors for RDD Japan.
Volunteer Participation
JCR employees will participate in volunteer activities at the Universal Baseball area themed “Move and Play,” where assistive technologies enable children requiring medical care to enjoy sports through finger-controlled digital interaction.
Awareness Display
An educational panel explaining lysosomal storage disorders in a clear and accessible way will be displayed at multiple venues nationwide, including Tokyo Tower FOOT TOWN (2F), the RDD Tokyo venue (KABUTO ONE), the RDD Japan event at Yamaguchi Prefecture, and other official RDD event venues. The panel will also be available via the RDD Japan website.
(URL: https://rddjapan.info/2026/panel/ (in Japanese))
Across the JCR Group, employees support RDD through ongoing awareness initiatives, from displaying posters and wearing official RDD badges to participating in sporting events in company-designed apparel and other outreach activities that help raise awareness of rare diseases. This year, JCR and MEDIPAL HOLDINGS CORPORATION hosted an internal workshop featuring perspectives from patient families, using a Patient Journey Map1 to deepen employee understanding of rare diseases.
The spirit of RDD is a part of who we are at JCR. Alongside advancing R&D to develop new therapies, we remain equally committed to awareness, inclusion, and standing with patients living with rare diseases worldwide.
About Rare Disease Day (RDD)
Rare Disease Day, launched in 2008, has grown into the world’s largest awareness campaign for rare diseases, celebrated in over 100 countries. In Japan, RDD official events have been held since 2010. This year’s theme in Japan highlights Tomo ni, Sugosu. Tomo ni, Tsukuru. Toki ni, Warau.
(Spending time together. Creating together. Laughing sometimes.)
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 partners, patients and employees. We strive to expand the possibilities for patients while accelerating medical advancement at a global level.
References
1: A tool illustrating a patient’s journey through diagnosis and treatment.
Rare Disease Day global website: https://www.rarediseaseday.org/
RDD Japan website: https://rddjapan.info/
Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com