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Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Relief Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Relief Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Iduronicrin Genleukocel-T","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Immusoft Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Immusoft Corporation \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Immusoft Corporation \/ Inapplicable"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"SION-638","moa":"NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"OrbiMed Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Series B Financing","leadProduct":"SION-638","moa":"||NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Sionna Therapeutics \/ OrbiMed Advisors","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ OrbiMed Advisors"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pasithea Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"WuXi AppTec","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Agreement","leadProduct":"PAS-004","moa":"MEK1\/2 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pasithea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pasithea Therapeutics \/ WuXi AppTec","highestDevelopmentStatusID":"6","companyTruncated":"Pasithea Therapeutics \/ WuXi AppTec"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Inapplicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"KB407","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Inapplicable"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Novel Pharma","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Novel Pharma"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Novel Pharma","highestDevelopmentStatusID":"6","companyTruncated":"GC Biopharma \/ Novel Pharma"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Magnesium","moa":"||Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Inapplicable"},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Magnesium Potentiated Oxytocin","moa":"Oxytocin receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Tonix Pharmaceuticals Holding Corp","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Inapplicable"},{"orgOrder":0,"company":"Columbia University","sponsor":"Actinium pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabelled Compounds","year":"2025","type":"Inapplicable","leadProduct":"131-I Apamistmab","moa":"CD45","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Columbia University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Columbia University \/ Actinium pharmaceuticals","highestDevelopmentStatusID":"6","companyTruncated":"Columbia University \/ Actinium pharmaceuticals"},{"orgOrder":0,"company":"Johns Hopkins University","sponsor":"Neurofibromatosis Therapeutic Acceleration Program | Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Talimogene Laherparepvec","moa":"Melanoma cell","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Johns Hopkins University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | Amgen Inc","highestDevelopmentStatusID":"6","companyTruncated":"Johns Hopkins University \/ Neurofibromatosis Therapeutic Acceleration Program | Amgen Inc"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Sionna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"SION-451","moa":"CFTR NBD1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sionna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Sionna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sionna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"EDK060","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"HG005","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"HuidaGene Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Restem","sponsor":"SOLVE FSHD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Umbilical Cord Lining-Derived Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Restem","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Restem \/ SOLVE FSHD","highestDevelopmentStatusID":"6","companyTruncated":"Restem \/ SOLVE FSHD"},{"orgOrder":0,"company":"Vascarta","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Curcumin","moa":"Albendazole monooxygenase (CYP3A4)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vascarta","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vascarta \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vascarta \/ Undisclosed"},{"orgOrder":0,"company":"Vascarta","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Agreement","leadProduct":"Curcumin","moa":"Albendazole monooxygenase (CYP3A4)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vascarta","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Vascarta \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"6","companyTruncated":"Vascarta \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"VCA-894A","moa":"IGHMBP2","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vanda Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Vanda Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Keros Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"KER-065","moa":"Myostatin\/Activin A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Keros Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Keros Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Keros Therapeutics \/ Undisclosed"}]

    Find Drugs for Genetic Disease in Phase I Clinical Development

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
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                            • 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) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 05, 2024

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            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) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 03, 2024

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Sanofi

                            France
                            CPhI WW Frankfurt
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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) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously R...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            November 24, 2023

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            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) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy ...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 22, 2023

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            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) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Excretion Balance, Pharmacokinetics, and Metabolism Following of [14C]-Venglustat Administration in Healthy Ma...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 14, 2022

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            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) : SAR439459

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            February 09, 2022

                            Lead Product(s) : SAR439459

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            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) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctoco...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            September 13, 2021

                            Lead Product(s) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            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) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type ...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 25, 2021

                            Lead Product(s) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Axplora

                            Germany
                            PREP
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothAxplora is your partner of choice for complex APIs.

                            Lead Product(s) : LYS-GM101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            Novasep and Lysogene Announce their New Collaboration for Development and Production of GM1 Gangliosidosis Gen...

                            Details : With this collaboration, the two companies consolidate their long-lasting partnership initiated with the development and manufacturing of Lysogene's lead gene therapy product, LYS-SAF302, currently in clinical phase 2/3.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            May 26, 2020

                            Lead Product(s) : LYS-GM101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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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 I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Effect of Venglustat in Patients With Renal Impairment

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            September 27, 2018

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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