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Shanghai Jiaotong University School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd.","highestDevelopmentStatusID":"6","companyTruncated":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd."},{"orgOrder":0,"company":"Shanghai Jiaotong University School of Medicine","sponsor":"Shanghai Vitalgen BioPharma Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VGN-R09b","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai Jiaotong University School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd.","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai Jiaotong University School of Medicine \/ Shanghai Vitalgen BioPharma Co., Ltd."},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"CAS 830-96-6","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VX-828","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":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"VX-828","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":"Zhongmou Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ZM-01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Zhongmou Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zhongmou Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Zhongmou Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Zhongmou Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ZM-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Zhongmou Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Zhongmou Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Zhongmou Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Shenzhen Hemogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"\u0392-Globin Restored Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shenzhen Hemogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shenzhen Hemogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shenzhen Hemogen \/ Undisclosed"},{"orgOrder":0,"company":"Shenzhen Hemogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"\u0392-Globin Restored Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shenzhen Hemogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shenzhen Hemogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shenzhen Hemogen \/ Undisclosed"},{"orgOrder":0,"company":"Shenzhen Hemogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"\u0392-Globin Restored Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shenzhen Hemogen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shenzhen Hemogen \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shenzhen Hemogen \/ Undisclosed"},{"orgOrder":0,"company":"Mission Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"MTX652","moa":"USP-30","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Mission Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Mission Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Mission Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"University of California, San Diego","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2011","type":"Inapplicable","leadProduct":"Ranibizumab","moa":"Vascular endothelial growth factor A","graph1":"Genetic Disease","graph2":"Phase I","graph3":"University of California, San Diego","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"University of California, San Diego \/ Genentech","highestDevelopmentStatusID":"6","companyTruncated":"University of California, San Diego \/ Genentech"},{"orgOrder":0,"company":"Autifony Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"AUT00201","moa":"VGKCs","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Autifony Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Autifony Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Autifony Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Radiolabeled Compound","year":"2012","type":"Inapplicable","leadProduct":"14-C AT1001","moa":"Zonulin","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Hitachi Global Life Solutions","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Agreement","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Hitachi Global Life Solutions","highestDevelopmentStatusID":"6","companyTruncated":"Elixirgen Therapeutics \/ Hitachi Global Life Solutions"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"INV-347","moa":"CB1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Shattuck Labs","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"SL-325","moa":"DR3","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shattuck Labs","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Shattuck Labs \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Shattuck Labs \/ Undisclosed"},{"orgOrder":0,"company":"Pharmbio Korea","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Levodropropizine","moa":"C-fiber sensory nerves","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Pharmbio Korea","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharmbio Korea \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Pharmbio Korea \/ Undisclosed"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"CHF6467","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chiesi Group \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Chiesi Group \/ Undisclosed"},{"orgOrder":0,"company":"CorrectSequence Therapeutics","sponsor":"The First Affiliated Hospital of Anhui Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"CS-121","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"CorrectSequence Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CorrectSequence Therapeutics \/ The First Affiliated Hospital of Anhui Medical University","highestDevelopmentStatusID":"6","companyTruncated":"CorrectSequence Therapeutics \/ The First Affiliated Hospital of Anhui Medical University"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SGT-212","moa":"FXN","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Solid Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Solid Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Solid Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Jemincare","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Amiodarone Hydrochloride","moa":"HERG","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Jemincare","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Jemincare \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Jemincare \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Cagrilintide","moa":"GLP-1 receptor","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"DISC-3405","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Disc medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Disc medicine \/ Undisclosed"},{"orgOrder":0,"company":"Shanghai Siponuoyin Biotechnology Co Ltd","sponsor":"Shanghai Children's Medical Center","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"SPOT-mRNA03","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Shanghai Siponuoyin Biotechnology Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Shanghai Siponuoyin Biotechnology Co Ltd \/ Shanghai Children's Medical Center","highestDevelopmentStatusID":"6","companyTruncated":"Shanghai Siponuoyin Biotechnology Co Ltd \/ Shanghai Children's Medical Center"},{"orgOrder":0,"company":"Beijing Inno Medicine","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"YN001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Beijing Inno Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Beijing Inno Medicine \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Beijing Inno Medicine \/ Undisclosed"},{"orgOrder":0,"company":"Angelini Pharma","sponsor":"Cross Research S.A.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Benzydamine Hydrochloride","moa":"Cyclooxygenase","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Angelini Pharma","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Angelini Pharma \/ Cross Research S.A.","highestDevelopmentStatusID":"6","companyTruncated":"Angelini Pharma \/ Cross Research S.A."},{"orgOrder":0,"company":"Tonix Pharmaceuticals Holding Corp","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2025","type":"Inapplicable","leadProduct":"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":"Spray","sponsorNew":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Tonix Pharmaceuticals Holding Corp \/ Undisclosed"},{"orgOrder":0,"company":"Karuna Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Xanomeline","moa":"Muscarinic acetylcholine receptor M1 | Muscarinic acetylcholine receptor M4","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Karuna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Karuna Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Karuna Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Anebulo Pharmaceuticals","sponsor":"National Institute on Drug Abuse","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Controlled Substance","year":"2025","type":"Inapplicable","leadProduct":"Selonabant","moa":"CNR1","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Anebulo Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Anebulo Pharmaceuticals \/ National Institute on Drug Abuse","highestDevelopmentStatusID":"6","companyTruncated":"Anebulo Pharmaceuticals \/ National Institute on Drug Abuse"}]

    Find Drugs for Genetic Disease in Phase I Clinical Development in UNITED STATES

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
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                            • fda
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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 : Undisclosed

                            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 : Eliglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Gaucher Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 05, 2024

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            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 : Undisclosed

                            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 : Eliglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Gaucher Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 03, 2024

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            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) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            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 : Fitusiran is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemophilia A.

                            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 : Undisclosed

                            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 : Undisclosed

                            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 : Eliglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Gaucher Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 22, 2023

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

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

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

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            February 14, 2022

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

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

                            Details : SAR439459 is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Osteogenesis Imperfecta.

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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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 : Undisclosed

                            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 : Efanesoctocog Alfa is a Protein drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemophilia A.

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            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 : Undisclosed

                            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 : Efanesoctocog Alfa is a Protein drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of von Willebrand Diseases.

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Axplora

                            Germany
                            CPhI WW Frankfurt
                            Booth #11.0B48
                            • 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

                            DEALS_DEV arrow-down

                            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 & 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

                            Axplora CB

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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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Effect of Venglustat in Patients With Renal Impairment

                            Details : Venglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 27, 2018

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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