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    [{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Olipudase Alfa","moa":"Sphingomyelin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"8","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Theravia","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Theravia \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Theravia \/ Inapplicable"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Vonafexor","moa":"Bile acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enyo Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Enyo Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Enyo Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Theravia","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Theravia","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Theravia \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Theravia \/ Inapplicable"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"Vesalius Biocapital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2025","type":"Series C Financing","leadProduct":"Vonafexor","moa":"Bile acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enyo Pharma","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Enyo Pharma \/ Vesalius Biocapital","highestDevelopmentStatusID":"8","companyTruncated":"Enyo Pharma \/ Vesalius Biocapital"},{"orgOrder":0,"company":"Orphalan","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Orphalan \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphalan \/ Inapplicable"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"OrbiMed Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2024","type":"Series C Financing","leadProduct":"Vonafexor","moa":"Bile acid receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Enyo Pharma","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Enyo Pharma \/ OrbiMed Advisors","highestDevelopmentStatusID":"8","companyTruncated":"Enyo Pharma \/ OrbiMed Advisors"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SAR447537","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SAR447537","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Orphalan","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Trientine Hydrochloride","moa":"Copper","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphalan","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orphalan \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Orphalan \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sp...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            April 29, 2025

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            June 09, 2023

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            May 12, 2023

                            Lead Product(s) : SAR447537

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Sanofi

                            France
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                            Attending
                            • fda
                            • EDQM
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Tesevatinib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            June 29, 2017

                            Lead Product(s) : Tesevatinib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patie...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            January 12, 2017

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Lademirsen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            August 04, 2016

                            Lead Product(s) : Lademirsen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Sanofi

                            France
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                            • fda
                            • EDQM
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monother...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 25, 2016

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Tesevatinib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Long-Term Treatment and Follow up of Subjects Completing 24 Months of Treatment With Tesevatinib on Study KD01...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 26, 2015

                            Lead Product(s) : Tesevatinib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Na...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 03, 2015

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 29, 2014

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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