JCR Pharmaceuticals celebrates its 50th anniversary of advancing science and changing lives
Hyogo, Japan – September 12, 2025 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced that it will celebrate its 50th anniversary on September 13, 2025, marking five decades of advancing science and redefining possibilities for patients living with complex healthcare challenges. The company expresses its deep gratitude to patients, families, partners, and stakeholders worldwide who have been part of its journey.
Founded in 1975, JCR has grown from a pioneering biotech in Japan into a global biopharmaceutical company, with operations spanning the United States, Europe, and South America. Guided by its corporate philosophy — to create treatments that go beyond rare disease to solve the world’s most complex healthcare challenges — JCR has delivered transformative therapies by leveraging breakthrough platforms in regenerative medicine, recombinant protein engineering, and gene therapy.
“This milestone isn’t just about looking back at what we’ve achieved; it’s also about reaffirming our commitment to patients and charting an even bolder course for the future,” said Shin Ashida, Chairman, President, and CEO of JCR Pharmaceuticals. “At JCR, we have always prided ourselves on cutting-edge science and pushing boundaries to develop new and innovative technologies with the goal of developing treatments for complex healthcare challenges for those who need them most. We have been and will always be committed to working closely with patients, families, and physicians to inform our research and development programs so that our treatments make a real difference. Our mission is simple – to give people living with complex disease hope of a better future, and we look forward to continuing on this mission.”
To commemorate the anniversary, JCR will launch a series of initiatives throughout the year, including newspaper and transit campaigns, a dedicated anniversary logo and key creative, and a special webpage that highlights the company’s history, the people behind its innovations, and its vision for the decades ahead.
Visit the anniversary page here:
https://www.jcrpharm.co.jp/50th/
About the Anniversary Key Creative and Logo
The key creative features a beam of light illuminating JCR’s research and development journey — a symbol of the hope the company strives to bring to patients and communities — while the streamlined, linear design of the anniversary logo expresses 50 years of progress driven by a steadfast focus on patients.
As JCR enters its next chapter, the company remains steadfast in its commitment to stand with every patient, tackle the toughest challenges in medicine, and create transformative therapies that change lives.
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 III A 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. For more information, please visit JCR’s global website:
https://jcrpharm.com/
Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com
MEDIPAL HOLDINGS and JCR Pharmaceuticals sign global licensing and Japan co-development agreement for JR-479, a novel therapy for GM2 gangliosidosis
Tokyo and Hyogo, Japan – August 29, 2025 – MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the two companies have signed an exclusive global licensing deal and a co-development and commercialization partnership in Japan for JR-479, an investigational therapy for GM2 gangliosidosis, an ultra-rare lysosomal storage disorder.
GM2 gangliosidoses are a group of rare, inherited disorders caused by genetic mutations that prevent the normal breakdown of GM2 gangliosides in lysosomes, leading to their buildup, especially in neurons. Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis depending on the specific mutation. GM2 gangliosidosis affects an estimated one in 300,000 people worldwide and is considered an ultra-rare, life-threatening disorder for which no approved treatments currently exist.
JR-479 is a novel drug candidate developed with JCR’s proprietary J-Brain Cargo® platform, clinically validated through IZCARGO® for I.V. infusion 10mg, which gained approval in Japan in 2021 for the treatment of a lysosomal storage disorder. In preclinical studies,¹ JR-479 was delivered throughout the body, including to the central nervous system, reducing disease-related substrates and extending survival in animal models.
Under the global licensing deal, MEDIPAL will have exclusive worldwide rights (excluding Japan) to develop, manufacture, and commercialize JR-479, with sub-licensing rights. In Japan, through the co-development partnership, MEDIPAL will handle clinical trial logistics, support disease awareness, and assist with operations, while JCR leads commercialization and records domestic revenue. JCR will also receive an upfront payment, and royalties on overseas sales.
This agreement adds JR-479 to the strategic collaboration between MEDIPAL and JCR, launched in October 2022, to develop treatments for ultra-rare diseases. It follows JR-471 for fucosidosis² and JR-446 for mucopolysaccharidosis type IIIB,³ making JR-479 the third pipeline in the partnership.
Both companies have already factored the financial impact of this agreement into their consolidated forecasts for the fiscal year ending March 31, 2026.
About the J-Brain Cargo® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology J-Brain Cargo®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a rare lysosomal storage disorder.
About lysosomal storage diseases
Lysosomal storage diseases are genetic disorders in which certain enzymes or proteins that remove unwanted substances from cells do not work as they should. When that happens, those substances build up inside the lysosomes and damage cells and tissues. The symptoms differ depending on what builds up, and many of these conditions also affect the central nervous system.
About GM2 Gangliosidosis (Tay-Sachs, Sandhoff disease)
GM2 gangliosidosis is a group of rare, inherited lysosomal storage disorders caused by deficiency of β-hexosaminidase A (Hex A), an enzyme consisting of α and β subunits. Depending on whether the α- or β- subunit is deficient, GM2 gangliosidosis is classified as Tay-Sachs or Sandhoff disease, respectively. Deficiency of Hex A results in the progressive accumulation of GM2 gangliosides and other glycolipids, which leads to severe neurodegeneration. In severe cases, nearly half of affected children do not survive past the age of three.⁴ Currently, there are no approved treatments, highlighting the urgent need for new therapies.
About MEDIPAL HOLDINGS CORPORATION
MEDIPAL HOLDINGS CORPORATION is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit https://www.medipal.co.jp/english/.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. is a global specialty pharmaceutical company dedicated that develops treatments that go beyond rare diseases to solve the world’s most complex healthcare challenges. JCR continues to build upon our 50-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. JCR’s innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more.
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.
References
1. Press release on research presentations at WORLDSymposium™ 2025 (February 5, 2025).
2. Press release on the exclusive negotiation rights for global commercialization targeting four ultra-rare diseases and the licensing contract for the fucosidosis therapeutic between MEDIPAL and JCR (October 31, 2022).
3. Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).
4. Bley AE, et al. Natural history of infantile G(M2) gangliosidosis. Pediatrics. 2011;128(5): e1233-1241.
Contacts
MEDIPAL HOLDINGS CORPORATION
Public Relations Department
TEL: (+81)-3-3517-5171
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
E-mail：ir-info@jp.jcrpharm.com