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[{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"General Atlantic","pharmaFlowCategory":"D","amount":"$98.0 million","upfrontCash":"Undisclosed","newsHeadline":"CANbridge Pharmaceuticals Completes US$98 Million Series D Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Tianjin Tiancheng Pharma. Co., Ltd.","sponsor":"Grifols International","pharmaFlowCategory":"D","amount":"$1,281.5 million","upfrontCash":"Undisclosed","newsHeadline":"Grifols Acquires Tiancheng Pharmaceutical Holdings, the Largest Shareholder of Biotest, to Increase Patients\u2019 Access to Plasma Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Tianjin Tiancheng Pharma. Co., Ltd."},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Commercialization of Gene Therapy is coming soon: Neurophth Completes Patient Enrollment for Phase III clinical trial for the gene therapy treatment of LHON","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurophth Therapeutics"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Belief BioMed Announces a Key Milestone of Dosing Completion for All Subjects in its Registrational Clinical Trial of BBM-H901","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Belief BioMed"}]

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            BBM-H901 is an adeno-associated virus (AAV) gene therapy, which is given to introduce the human factor Ⅸ (FⅨ) gene into the body of hemophilia B patient, thus maintaining the levels of coagulation factors.

            Lead Product(s): BBM-H901

            Therapeutic Area: Genetic Disease Product Name: BBM-H901

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 23, 2023

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            NR082 (rAAV2-ND4), a novel recombinant AAV2, containing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 gene which delivers the correct genes to the patients' damaged optic ganglion cells. It is being developed for LHON associated with mtND4 mutations.

            Lead Product(s): NR082

            Therapeutic Area: Genetic Disease Product Name: NR082

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 22, 2023

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            This acquisition will significantly reinforce Grifols’ industry capabilities by enhancing its plasmaderived medicines access, pipeline Biotest is leading clinical trials on plasma-derived fibrinogen (BT524) to treat congenital and acquired disorders.

            Lead Product(s): BT524

            Therapeutic Area: Genetic Disease Product Name: BT524

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Grifols International

            Deal Size: $1,281.5 million Upfront Cash: Undisclosed

            Deal Type: Acquisition September 17, 2021

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            The proceeds, will be used to accelerate and expand the CANbridge rare disease pipeline through internal development and external partnerships, further build CANbridge’s commercial infrastructure.

            Lead Product(s): Idursulfase beta

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: General Atlantic

            Deal Size: $98.0 million Upfront Cash: Undisclosed

            Deal Type: Series D Financing February 18, 2020

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