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    [{"orgOrder":0,"company":"Azafaros","sponsor":"Forbion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Small molecule","year":"2020","type":"Series A Financing","leadProduct":"AZ-3102","moa":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Azafaros","amount2":0.029999999999999999,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Oral","sponsorNew":"Azafaros \/ Forbion","highestDevelopmentStatusID":"1","companyTruncated":"Azafaros \/ Forbion"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"AZ-3102","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic 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Disease","graph2":"Phase I","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Azafaros \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Not Applicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"AZ-3102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Azafaros \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Azafaros \/ Not Applicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not 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Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Not Applicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Nizubaglustat","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Azafaros \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Not Applicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Nizubaglustat","moa":"","graph1":"Genetic Disease","graph2":"Phase 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                            01

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Announces Positive Phase 2 Data with Nizubaglustat in GM2 and Niemann-Pick

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor, being evaluated for the treatment of GM2 Gangliosidosis and Type C Niemann-Pick Disease.

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 16, 2024

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            02

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Completes Phase 2 RAINBOW Study for Nizubaglustat in Rare Diseases

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor. Currently, it has completed the ongoing Phase II clinical trial studies for GM2 Gangliosidosis and Type C Niemann-Pick Disease.

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 12, 2024

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros™ Phase 2 RAINBOW study, Evaluating Nizubaglustat in GM2 and NPC Patients, is Now Fully Enrolled

                            Details : AZ-3102 (nizubaglustat) is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick diseas...

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            December 11, 2023

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            Azafaros

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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Pat...

                            Details : AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            June 08, 2023

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            Azafaros

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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Receives FDA’s IND Clearance and Fast Track Designation

                            Details : AZ-3102 is a, novel small molecule, candidate developed for Lysosomal Storage Disorders with neurological involvement. AZ-3102 is an oral, brain penetrant azasugar, having dual mode of action by inhibiting two key enzymes which modulate the metabolism of...

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            January 05, 2023

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            Azafaros

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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease

                            Details : AZ‑3102, an orally available, small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism, is designed to selectively inhibit two enzymes involved in glycolipid metabolism aims to reduce toxic ...

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 24, 2022

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Azafaros

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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Ly...

                            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...

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            February 14, 2022

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            Azafaros

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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis

                            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...

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            February 01, 2022

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            Azafaros

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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Azafaros Enters Clinical Stage with AZ-3102, An Oral Small Molecule Being Developed for Rare Neurogenetic Diso...

                            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...

                            Brand Name : AZ-3102

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            April 06, 2021

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            Azafaros

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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Forbion

                            Deal Size : $28.0 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Azafaros Completes EUR 25 Million Series A Financing

                            Details : The financing helps Azafaros to build its organization and expand its executive team with passionate and experienced people and to advance its rare metabolic disorders pipeline.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            February 06, 2020

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Forbion

                            Deal Size : $28.0 million

                            Deal Type : Series A Financing

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