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              Cycle Pharmaceuticals

              • Development Update

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              UNITED KINGDOM United Kingdom

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              World Dairy Expo

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              US FDA Grants Once Daily Dosing Option for NITYR (nitisinone) Tablets for HT-1 Patients

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              Lead Product(s): Nitisinone

              Therapeutic Area: Genetic Disease Product Name: Nityr

              Highest Development Status: Approved Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable September 14, 2020

              Details:

              NITYR® is indicated for the treatment of patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

              Bluebird bio

              • Development Update

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              UNITED KINGDOM United Kingdom

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              World Dairy Expo

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              Majority of Evaluable Patients Across Genotypes Achieve Transfusion Independence and Maintain It with Near-Normal Hemoglobin Levels in Phase...

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              Lead Product(s): Betibeglogene autotemcel

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

              Highest Development Status: Approved Product Type: Cell and Gene therapy

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable June 12, 2020

              Details:

              89% of evaluable patients (17/19) with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total hemoglobin (Hb) level in HGB-207.

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