[{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","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":"Oral","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","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":"Oral","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Inapplicable"}]

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                          01

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          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 : AZ-3102

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 14, 2022

                          Lead Product(s) : Nizubaglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : The ODD for AZ-3102, selectively inhibit two enzymes involved in glycolipid metabolism GCS and GbA2 for the treatment ofGM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survi...

                          Product Name : AZ-3102

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 02, 2022

                          Lead Product(s) : Nizubaglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : Phase 1 clinical study investigates safety and tolerability, pharmacokinetics and pharmacodynamic effects of first-in-class azasugar AZ-3102 in healthy subjects. AZ-3102 is being initially developed for the treatment of patients suffering from GM1 and GM...

                          Product Name : AZ-3102

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          June 04, 2021

                          Lead Product(s) : Nizubaglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

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

                          Product Name : AMT-130

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 27, 2020

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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