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              JCR Pharmaceuticals

              • Development Update

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              JAPAN Japan

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              JCR Pharmaceuticals Completes Phase 1/2 Clinical Trial Notification of JR-171 for mucopolysaccharidosis type I

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              Lead Product(s): alpha L Idosiduronase

              Therapeutic Area: Genetic Disease Product Name: JR-171

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable July 31, 2020

              Details:

              JR-171 is a blood-brain-barrier (BBB)-penetrating recombinant α-L-iduronidase, the product candidate for the treatment of patients with MPS I, to which J-Brain Cargo®, JCR’s proprietary BBB penetration technology, is applied.

              Hitachi Chemicals

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              Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma ink Clinical and Commercial Manufacturing Pact with Bluebird Bio

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              Lead Product(s): LentiGlobin

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

              Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

              Partner/Sponsor/Collaborator: Bluebird bio

              Deal Size: Undisclosed Upfront Cash: Undisclosed

              Deal Type: Agreement May 21, 2020

              Details:

              The two subsidiaries, Hitachi Chemical Advanced Therapeutics Solutions (HCATS) and Apceth Biopharma, will be in charge of late-clinical development and manufacturing of LentiGlobin at facilities in Germany (Apceth) and the U.S. (HCATS).

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