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    [{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Peking Union Medical College Hospital","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 1\/2 Trial for the Treatment of Gaucher Disease in China","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 2 Trial for the Treatment of Gaucher Disease in China","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"OBiO Technology","sponsor":"Chinagene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"OBiO and Chinagene Reached A Strategic Agreement Of Commercial Manufacturing Services For Gene Therapy Products","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"OBiO Technology"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Announces First Patient Dosed in Phase I\/II Clinical Trial of Second Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 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 Successfully Completed Dosing of First Subject in the Registrational Clinical Trial for Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Belief BioMed"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Announces Completion of Patient Enrollment for Opvika\u00ae Phase I\/II Clinical Trial in the U.S.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurophth Therapeutics"}]

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              Neurophth Therapeutics

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              Neurophth Announces Completion of Patient Enrollment for Opvika® Phase I/II Clinical Trial in the U.S.

              Details:

              Opvika (Esonadogene Imvoparvovec) is a MT-ND4 modulator. It is being evaluated for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON).

              Lead Product(s): Esonadogene Imvoparvovec

              Therapeutic Area: Genetic Disease Product Name: Opvika

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

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 19, 2024

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              Belief BioMed

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              Belief BioMed Successfully Completed Dosing of First Subject in the Registrational Clinical Trial for Hemophilia A

              Details:

              BBM-H803 is an AAV-based gene therapy. It is administered intravenously to deliver the coagulation factor Ⅷ gene into the body of patients with hemophilia A,

              Lead Product(s): BBM-H803

              Therapeutic Area: Genetic Disease Product Name: BBM-H803

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

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 03, 2024

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              Neurophth Therapeutics

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              Neurophth Announces First Patient Dosed in Phase I/II Clinical Trial of Second Gene Therapy

              Details:

              NFS-02 is Neurophth’s second gene therapy, currently evaluating in multi-region, multi-center Phase I/II clinical trial for the treatment of Leber hereditary optic neuropathy caused by ND1 mutation.

              Lead Product(s): NFS-02

              Therapeutic Area: Genetic Disease Product Name: NFS-02

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

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable August 17, 2023

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              OBiO Technology

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              OBiO and Chinagene Reached A Strategic Agreement Of Commercial Manufacturing Services For Gene Therapy Products

              Details:

              The collaboration aims to accelerate the commercialization of CHINAGENE's ophthalmic gene therapy product ZVS101e. Also OBiO will provide holistic contract development and manufacturing services to CHINAGENE for their gene therapy products.

              Lead Product(s): ZVS101e

              Therapeutic Area: Genetic Disease Product Name: ZVS101e

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

              Partner/Sponsor/Collaborator: Chinagene

              Deal Size: Undisclosed Upfront Cash: Undisclosed

              Deal Type: Collaboration April 29, 2023

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              CANbridge Pharmaceuticals

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              First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 2 Trial for the Treatment of Gaucher Disease in China

              Details:

              CAN103 is a recombinant human glucocerebrosidase enzyme replacement therapy that is being developed to treat Gaucher disease (GD) Types I and III, which are the chronic non-neuronopathic and neuronopathic forms of the disease that constitute the majority of patients.

              Lead Product(s): CAN103

              Therapeutic Area: Genetic Disease Product Name: CAN103

              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 January 18, 2023

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              CANbridge Pharmaceuticals

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              First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 1/2 Trial for the Treatment of Gaucher Disease in China

              Details:

              CAN103 is a recombinant human glucocerebrosidase enzyme replacement therapy that is being developed to treat Gaucher disease (GD) Types I and III, which are the chronic non-neuronopathic and neuronopathic forms of the disease that constitute the majority of patients.

              Lead Product(s): CAN103

              Therapeutic Area: Genetic Disease Product Name: CAN103

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

              Partner/Sponsor/Collaborator: Peking Union Medical College Hospital

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable July 20, 2022

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              Minakem offers CDMO services for API & HPAPI, generics, regulatory expertise, track record performance & FDA & GMP certifications.
              ChemWerth works in generic API development & supply, non-infringement patent strategy development and regulatory support.
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