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Tauli","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Tridocosahexaenoine","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Corporacion Parc Tauli","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Corporacion Parc Tauli \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Corporacion Parc Tauli \/ Inapplicable"},{"orgOrder":0,"company":"Western Sydney Local Health District","sponsor":"Westmead Institute for Medical Research","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Beetroot","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Western Sydney Local Health District","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Western Sydney Local Health District \/ Westmead Institute for Medical Research","highestDevelopmentStatusID":"1","companyTruncated":"Western Sydney Local Health District \/ Westmead Institute for Medical Research"},{"orgOrder":0,"company":"Seventh Medical Center of PLA General Hospital","sponsor":"GeneCradle Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"GC301","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Seventh Medical Center of PLA General Hospital","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Seventh Medical Center of PLA General Hospital \/ GeneCradle Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Seventh Medical Center of PLA General Hospital \/ GeneCradle Therapeutics"},{"orgOrder":0,"company":"He Huang","sponsor":"Lingyi Biotech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"LY-M001","moa":"SARS-CoV-2 spike protein","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"He Huang","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"He Huang \/ Lingyi Biotech","highestDevelopmentStatusID":"1","companyTruncated":"He Huang \/ Lingyi Biotech"},{"orgOrder":0,"company":"Ajinomoto Company","sponsor":"Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Glytactin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Ajinomoto Company","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ajinomoto Company \/ Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust","highestDevelopmentStatusID":"1","companyTruncated":"Ajinomoto Company \/ Birmingham Children's Hospital | Great Ormond Street Hospital for Children NHS Foundation Trust"},{"orgOrder":0,"company":"ALPS Biotech CO. LTD","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"Antrodia Cinnamomea","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"ALPS Biotech CO. LTD","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ALPS Biotech CO. LTD \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"ALPS Biotech CO. LTD \/ Inapplicable"},{"orgOrder":0,"company":"Genecombio Ltd.","sponsor":"The Children's Hospital of Zhejiang University School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GCB-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Genecombio Ltd.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genecombio Ltd. \/ The Children's Hospital of Zhejiang University School of Medicine","highestDevelopmentStatusID":"1","companyTruncated":"Genecombio Ltd. \/ The Children's Hospital of Zhejiang University School of Medicine"}]

Find Novel Drugs for Genetic Disease under Development

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                          Top Deals by Deal Size (USD bn)

                          01

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 19, 2024

                          Lead Product(s) : Fosigotifator

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          SCI Medicinal Chemistry
                          Not Confirmed
                          SCI Medicinal Chemistry
                          Not Confirmed

                          Details : Advanz secures exclusive rights from Alvotech to commercialize three biosimilar candidates, Ilaris (canakinumab), Kesimpta (ofatumumab) and an undiclosed biosimilar in Europe.

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Undisclosed

                          May 28, 2025

                          Lead Product(s) : Canakinumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Advanz Pharma

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          03

                          SCI Medicinal Chemistry
                          Not Confirmed
                          SCI Medicinal Chemistry
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          May 02, 2025

                          Lead Product(s) : TAK-881

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Nutricia Inc

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                          SCI Medicinal Chemistry
                          Not Confirmed

                          Nutricia Inc

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                          SCI Medicinal Chemistry
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Dietary Supplement

                          Upfront Cash : Inapplicable

                          April 23, 2025

                          Lead Product(s) : GMP Intervention Product

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Applied Science & Performance Institute

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                          SCI Medicinal Chemistry
                          Not Confirmed

                          Applied Science & Performance Institute

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                          SCI Medicinal Chemistry
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Plant Extract/Herbal

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : CAS 501-94-0

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          SCI Medicinal Chemistry
                          Not Confirmed
                          SCI Medicinal Chemistry
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          March 14, 2025

                          Lead Product(s) : Apitegromab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          SCI Medicinal Chemistry
                          Not Confirmed
                          SCI Medicinal Chemistry
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          January 28, 2025

                          Lead Product(s) : Plozasiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Genecombio Ltd.

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                          SCI Medicinal Chemistry
                          Not Confirmed

                          Genecombio Ltd.

                          Country arrow
                          SCI Medicinal Chemistry
                          Not Confirmed

                          Lead Product(s) : GCB-001

                          Therapeutic Area : Genetic Disease

                          Study Phase : Undisclosed

                          Sponsor : The Children's Hospital of Zhejiang University School of Medicine

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          January 13, 2025

                          Lead Product(s) : GCB-001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : The Children's Hospital of Zhejiang University School of Medicine

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Institute of Hematology & Blood Diseases Hospital, China

                          Country arrow
                          SCI Medicinal Chemistry
                          Not Confirmed

                          Institute of Hematology & Blood Diseases Hospital, China

                          Country arrow
                          SCI Medicinal Chemistry
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          January 10, 2025

                          Lead Product(s) : CM-336

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Connoya Biomedical Technology

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          University Hospital, Montpellier

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                          SCI Medicinal Chemistry
                          Not Confirmed

                          University Hospital, Montpellier

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                          SCI Medicinal Chemistry
                          Not Confirmed

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Study Phase : Undisclosed

                          Sponsor : Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          December 12, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Institut de Recherche en Infectiologie de Montpellier (CNRS) | A*STAR

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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