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JCR Pharmaceuticals Co., Ltd. announced today that Takeda Pharmaceutical Co., Ltd. (“Takeda”) decided to discontinue their collaboration with JCR to develop gene therapies using adeno-associated viruses (AAV) combined with the JCR J-Brain Cargo ® Technology.

JCR Pharmaceuticals Announces Achievement of Milestone Using J-Brain Cargo ® Technology for Neurodegenerative Disease in Research Collaboration with Alexion.

JCR Pharmaceuticals Co., Ltd. announced today that it will present six presentations at the 20th Annual WORLD Symposium ™ 2024, to be held February 4-9, 2024 in San Diego, Calif.

Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) today announced that it has entered into a joint research agreement with JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) on joint evaluation of the drug delivery technology of the gene therapy to the central nervous system (CNS) assuming CNS diseases as a target.

JCR Pharmaceuticals’ Research Presentations at WORLD Symposium ™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders.

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida: “JCR”) announces that it has signed a Research Collaboration, Option and License Agreement with Alexion, AstraZeneca Rare Disease (“Alexion”) for the development of novel oligonucleotide therapeutics to support targeted delivery to certain tissues or organs using J-Brain Cargo®, JCR’s proprietary blood-brain barrier (BBB)-penetrating technology.

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that the first patient in the Phase I/II clinical trial with Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome Type A) has been dosed with JR‑441, a blood-brain barrier (BBB) penetrating form of heparan N-sulfatase that was developed using JCR’s proprietary J-Brain Cargo ® BBB-penetrating technology.

JCR Pharmaceuticals Co., Ltd. announced key results from the 52-week interim data of its global phase I/II study with JR-171 in individuals with mucopolysaccharidosis type I.

Thursday, February 29 is Rare Disease Day (“RDD”). RDD activities aim to raise awareness of rare diseases and to improve the quality of life of individuals with rare and intractable diseases through better diagnoses and treatments.

Angelini Pharma, part of the privately owned Angelini Industries, and JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that they entered into an exclusive global development and commercialization agreement for the development of novel biologic therapies that applies J-Brain Cargo®, blood-brain barrier penetrating technology, for the treatment of epilepsy.