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Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Voncento","moa":"von Willebrand factor | Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Chris Goss","sponsor":"Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Chris Goss","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chris Goss \/ Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington","highestDevelopmentStatusID":"11","companyTruncated":"Chris Goss \/ Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington"},{"orgOrder":0,"company":"Versiti","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"F10","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Versiti","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Versiti \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Versiti \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Michael Rafii, MD, PhD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Donanemab","moa":"Amyloid-beta A4 protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Eli Lilly \/ Michael Rafii, MD, PhD","highestDevelopmentStatusID":"11","companyTruncated":"Eli Lilly \/ Michael Rafii, MD, PhD"},{"orgOrder":0,"company":"Paratek Pharmaceuticals","sponsor":"Paul Beringer","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Omadacycline","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Paratek Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Paratek Pharmaceuticals \/ Paul Beringer","highestDevelopmentStatusID":"11","companyTruncated":"Paratek Pharmaceuticals \/ Paul Beringer"},{"orgOrder":0,"company":"Merck & Co","sponsor":"Joseph Kuti","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ceftolozane Sulfate","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Merck & Co","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Merck & Co \/ Joseph Kuti","highestDevelopmentStatusID":"11","companyTruncated":"Merck & Co \/ Joseph Kuti"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Ari Zimran","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Ari Zimran","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Ari Zimran"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Amy Tang","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Amy Tang","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Amy Tang"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Ala'a Sharara","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Sofosbuvir","moa":"Hepatitis C virus NS5B RNA-dependent RNA polymerase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ Ala'a Sharara","highestDevelopmentStatusID":"11","companyTruncated":"Gilead Sciences \/ Ala'a Sharara"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Linda Randolph","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Linda Randolph","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Linda Randolph"},{"orgOrder":0,"company":"H. Lundbeck AS","sponsor":"William Ondo, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Tetrabenazine","moa":"Synaptic vesicular amine transporter","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"H. Lundbeck AS","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pill","sponsorNew":"H. Lundbeck AS \/ William Ondo, MD","highestDevelopmentStatusID":"11","companyTruncated":"H. Lundbeck AS \/ William Ondo, MD"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            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 IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

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

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            April 24, 2025

                            Lead Product(s) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            August 31, 2023

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            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) : alpha L Idosiduronase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            China Post-marketing Surveillance (PMS) Study of Aldurazyme®

                            Details : Laronidase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Mucopolysaccharidosis I.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            November 26, 2021

                            Lead Product(s) : alpha L Idosiduronase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AbbVie Inc

                            U.S.A
                            ISPE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or ...

                            Details : Pancrelipase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 06, 2021

                            Lead Product(s) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            China Post-marketing Surveillance (PMS) Study of Fabrazyme®

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            September 23, 2021

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            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) : Imiglucerase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ...

                            Details : Imiglucerase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Gaucher Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            December 07, 2020

                            Lead Product(s) : Imiglucerase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Ver...

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 30, 2019

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            AbbVie Inc

                            U.S.A
                            ISPE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Compare US Marketed Creon Manufactured With a Modernized Process at an Alternate Manufacturing Site...

                            Details : Pancrelipase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            April 23, 2019

                            Lead Product(s) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

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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) : Factor VIII Fc Fusion Protein

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participa...

                            Details : Factor VIII Fc Fusion Protein is a Protein-drug Conjugate drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Protein-drug Conjugate

                            Upfront Cash : Inapplicable

                            March 28, 2017

                            Lead Product(s) : Factor VIII Fc Fusion Protein

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Swedish Orphan Biovitrum AB

                            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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsida...

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            July 26, 2012

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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