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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 October 30, 2020
Details:
In this study, MPS I patients will be treated with JR-171 for at most 12 weeks to evaluate the safety of JR-171 as the primary endpoint and exploratory efficacy and pharmacokinetics as secondary endpoints.
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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.
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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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