[{"orgOrder":0,"company":"Azafaros","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azafaros \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Undisclosed"},{"orgOrder":0,"company":"Azafaros","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azafaros \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Undisclosed"},{"orgOrder":0,"company":"Khondrion","sponsor":"University Ghent","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"KH176","moa":"mPGES-1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Khondrion","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Khondrion \/ University Ghent","highestDevelopmentStatusID":"6","companyTruncated":"Khondrion \/ University Ghent"},{"orgOrder":0,"company":"Argenx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Antibody, Unconjugated","year":"2024","type":"Inapplicable","leadProduct":"ARGX-119","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Argenx","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Argenx \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Argenx \/ Undisclosed"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : ARGX-119 is a Antibody drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Myasthenic Syndromes, Congenital.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          May 31, 2024

                          Lead Product(s) : ARGX-119

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Positive safety, tolerability and pharmacodynamics data from multiple dose levels in healthy volunteers support evaluation of AZ-3102, a small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolis...

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          February 14, 2022

                          Lead Product(s) : Nizubaglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Up to two years of follow-up in large transgenic huntington’s disease model demonstrates stable mHTT protein lowering.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          February 27, 2020

                          Lead Product(s) : Antimony pentoxide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : QR-010 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          September 30, 2015

                          Lead Product(s) : QR-010

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : European Commission

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : KH176 is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of MELAS Syndrome.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 09, 2015

                          Lead Product(s) : KH176

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : University Ghent

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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