[{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & 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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & 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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"AccurEdit Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & 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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"AccurEdit Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & 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":"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":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"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":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"CANbridge Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"TJ Biopharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Plonmarlimab","moa":"Granulocyte-macrophage colony-stimulating factor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"TJ Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TJ Biopharma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"TJ Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2017","type":"Inapplicable","leadProduct":"Pegylated Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","highestDevelopmentStatusID":"8","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2017","type":"Inapplicable","leadProduct":"Pegylated Somatropin","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of","highestDevelopmentStatusID":"8","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Tongji Hospital | The First Hospital of Jilin University | Affiliated Hospital of Jiangnan University | The First Affiliated Hospital with Nanjing Medical University | Shanghai Children's Hospital | The Children's Hospital of Zhejiang University School of"},{"orgOrder":0,"company":"Argo Biopharma","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Argo Biopharma \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai Kechow Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tunlametinib","moa":"ERK activator kinase (MEK)||Mitogen-activated protein kinase","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Shanghai Kechow Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Visen Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Peptide, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Visen Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Visen Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Peptide, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Visen Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Angitia Biopharmaceuticals","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Angitia Biopharmaceuticals \/ 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"},{"orgOrder":0,"company":"Akeso","sponsor":"AD Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Antibody, Unconjugated","year":"2019","type":"Inapplicable","leadProduct":"AK102","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Akeso","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Akeso \/ AD Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Akeso \/ AD Pharmaceuticals"},{"orgOrder":0,"company":"Connoya Biomedical Technology","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","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":"Jemincare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","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 \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Jemincare \/ Undisclosed"},{"orgOrder":0,"company":"Jemincare","sponsor":"Zhejiang Hangyu Pharmaceutical Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","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."}]

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                          01

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          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

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          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 & 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

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          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

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Plonmarlimab is a Antibody drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Lymphohistiocytosis, Hemophagocytic.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          June 24, 2025

                          Lead Product(s) : Plonmarlimab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          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 & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 28, 2025

                          Lead Product(s) : ART001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          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 & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 28, 2025

                          Lead Product(s) : ART001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : BW-20805 is a Oligonucleotide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Angioedemas, Hereditary.

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : JMKX003142 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          January 30, 2025

                          Lead Product(s) : JMKX003142

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : KN057 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Hemophilia A.

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          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, Unconjugated

                          Upfront Cash : Inapplicable

                          November 16, 2023

                          Lead Product(s) : Navepegritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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