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    [{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ART001","moa":"Amyloidogenic TTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AccurEdit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AccurEdit Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ART001","moa":"Amyloidogenic TTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AccurEdit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AccurEdit Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ART001","moa":"Amyloidogenic TTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AccurEdit Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AccurEdit Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Peking Union Medical College Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Peking Union Medical College Hospital","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Peking Union Medical College Hospital"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"glucocerebrosidase","moa":"Glucocerebrosidase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"Jemincare","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"JMKX003142","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jemincare","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jemincare \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Jemincare \/ Inapplicable"},{"orgOrder":0,"company":"Shanghai Kechow Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Tunlametinib","moa":"ERK activator kinase (MEK)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Shanghai Kechow Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Shanghai Kechow Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Shanghai Kechow Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Jemincare","sponsor":"Zhejiang Hangyu Pharmaceutical Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"JMKX003142","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Jemincare","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Jemincare \/ Zhejiang Hangyu Pharmaceutical Co., Ltd.","highestDevelopmentStatusID":"8","companyTruncated":"Jemincare \/ Zhejiang Hangyu Pharmaceutical Co., Ltd."},{"orgOrder":0,"company":"Visen Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Peptide","year":"2021","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Visen Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Visen Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Visen Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Visen Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Peptide","year":"2023","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Visen Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Visen Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Visen Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Angitia Biopharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AGA2115","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Angitia Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Angitia Biopharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Angitia Biopharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Argo Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"BW-20805","moa":"KLKB1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Argo Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Argo Biopharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Argo Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Connoya Biomedical Technology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"CM336","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Connoya Biomedical Technology","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Connoya Biomedical Technology \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Connoya Biomedical Technology \/ Undisclosed"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Equecabtagene Autoleucel","moa":"BCMA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"IASO Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IASO Bio \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"IASO Bio \/ Undisclosed"},{"orgOrder":0,"company":"Angitia Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"AGA2115","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Angitia Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Angitia Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Angitia Biopharmaceuticals \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Angitia Biopharmaceuticals

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            Angitia Biopharmaceuticals

                            China arrow
                            Cell Therapy
                            Not Confirmed
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                            Lead Product(s) : AGA2115

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            AGA2115 Trial in Adults with Osteogenesis Imperfecta COL1A1/COL1A2 Variations

                            Details : AGA2115 is a drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Osteogenesis Imperfecta.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            July 14, 2025

                            Lead Product(s) : AGA2115

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            IASO Bio

                            China arrow
                            Cell Therapy
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                            IASO Bio

                            China arrow
                            Cell Therapy
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : Equecabtagene Autoleucel

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Eque-cel CAR-T Therapy in Newly Diagnosed Severe AL Amyloidosis

                            Details : Equecabtagene Autoleucel is a Cell and Gene Therapy drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            July 09, 2025

                            Lead Product(s) : Equecabtagene Autoleucel

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Connoya Biomedical Technology

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            Connoya Biomedical Technology

                            China arrow
                            Cell Therapy
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : CM336

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy & Safety of CM336 Injection in Treatment of Light-Chain Amyloidosis

                            Details : CM336 is a drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            June 26, 2025

                            Lead Product(s) : CM336

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AccurEdit Therapeutics

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            AccurEdit Therapeutics

                            China arrow
                            Cell Therapy
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ART001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            AccurEdit's ART001 Becomes first Gene Editing Therapy in China with RMAT Status

                            Details : ART001 is the first gene editing product from China to receive an FDA RMAT designation, highlighting the FDA's recognition of its innovative nature and clinical potential.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 28, 2025

                            Lead Product(s) : ART001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            AccurEdit Therapeutics

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            AccurEdit Therapeutics

                            China arrow
                            Cell Therapy
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ART001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            AccurEdit Therapeutics' ART001 Becomes China's First Gene Editing Therapy to Receive FDA Regenerative Medicine...

                            Details : ART001 is an in vivo gene editing product delivered by LNP for the treatment of Transthyretin amyloidosis (ATTR).

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 28, 2025

                            Lead Product(s) : ART001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Argo Biopharma

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            Argo Biopharma

                            China arrow
                            Cell Therapy
                            Not Confirmed
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                            Lead Product(s) : BW-20805

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 2 in Adult Subjects With Hereditary Angioedema

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 26, 2025

                            Lead Product(s) : BW-20805

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Jemincare

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            Jemincare

                            China arrow
                            Cell Therapy
                            Not Confirmed
                            • fda
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                            Lead Product(s) : JMKX003142

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Trial of JMKX003142 in Participants With Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADP...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 30, 2025

                            Lead Product(s) : JMKX003142

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Alphamab Oncology

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            Alphamab Oncology

                            China arrow
                            Cell Therapy
                            Not Confirmed
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                            Lead Product(s) : KN057

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            KN057 Multiple Dose Study in Patients with Hemophilia a or Hemophilia B with or Without Inhibitors

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            December 24, 2024

                            Lead Product(s) : KN057

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Visen Pharmaceuticals

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            Visen Pharmaceuticals

                            China arrow
                            Cell Therapy
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Once-Weekly TransCon™ CNP Achieved Primary Efficacy Objective in Accomplish China Phase 2 Trial

                            Details : TransCon CNP (c-type natriuretic peptide prodrug) is a FGFR 3 inhibitor. It is being evaluated for the treatment of children with achondroplasia (ACH) aged 2 to 10 years.

                            Product Name : TransCon CNP

                            Product Type : Peptide

                            Upfront Cash : Inapplicable

                            November 16, 2023

                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            CANbridge Pharmaceuticals

                            China arrow
                            Cell Therapy
                            Not Confirmed

                            CANbridge Pharmaceuticals

                            China arrow
                            Cell Therapy
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : glucocerebrosidase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            CANbridge Announces Full Enrollment Reached in the Core Part of The CAN103 Phase 2 Trial for Gaucher Disease i...

                            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...

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 16, 2023

                            Lead Product(s) : glucocerebrosidase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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