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Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Fsio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Agreement","leadProduct":"Lumacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Fsio","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Fsio"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Lumacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex 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Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Oberland Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2021","type":"Financing","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Oberland Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Oberland Capital Management"},{"orgOrder":0,"company":"DEKA Research & Development","sponsor":"Zealand Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2021","type":"Collaboration","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"DEKA Research & Development","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"DEKA Research & Development \/ Zealand Pharma","highestDevelopmentStatusID":"10","companyTruncated":"DEKA Research & Development \/ Zealand Pharma"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Partnership","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ AnGes","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ AnGes"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"AnGes","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ AnGes","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ AnGes"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Acquisition","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"The Progeria Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Divestment","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"The Progeria Research Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small 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Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small 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Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Kaken Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Licensing Agreement","leadProduct":"Sebetralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"KalVista Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Oral Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Kaken Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"KalVista Pharmaceuticals \/ Kaken Pharmaceuticals"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avidity Biosciences \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"KYE Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KYE Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"KYE Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"KYE Pharmaceuticals \/ Inapplicable"}]

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                            01

                            AbbVie Inc

                            U.S.A
                            German Wound Congress
                            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) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Sionna Expands CF Pipeline with AbbVie License Agreement

                            Details : Under the terms of the agreement, Sionna will assume all development for ABBV-2222 (galicaftor), which is being evaluated in combination with navocaftor for the treatment of Cystic Fibrosis.

                            Product Name : ABBV-2222

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            July 16, 2024

                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            02

                            Sanofi

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

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Data Show Safety, Benefit, and Durability of Nexviazyme in Pompe Disease Patients

                            Details : Nexviazyme (avalglucosidase alfa) is a USFDA approved GAA stimulator enzyme replacement therapy. It is being evaluated for the treatment of treatment-naïve Infantile-onset Pompe disease.

                            Product Name : Nexviazyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            May 02, 2024

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
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                            • 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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Once-weekly ALTUVIIIO® Approved in Japan as a New Class of Factor VIII Therapy for Hemophilia A

                            Details : Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels, extending bleed protection in a once-weekly dose for people with haemophilia A.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            September 25, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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 III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Press Release: Two Fitusiran Phase 3 Studies Published in The Lancet and The Lancet Haematology Highlight Pote...

                            Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                            Product Name : SAR439774

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            April 04, 2023

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Phase 3 Data Presented At WORLD Symposium™ Reinforce Nexviazyme® (avalglucosidase alfa) As Potential Ne...

                            Details : Nexviazyme (avalglucosidase alfa) is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. It is being developed for treatment of people living with pompe disease.

                            Product Name : Nexviazyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            February 24, 2023

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            FDA Approves Once-Weekly ALTUVIIIO A New Class of Factor VIII Therapy for Hemophilia a That Offers Significant...

                            Details : Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophi...

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 23, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            NEJM Publishes Once-Weekly Efanesoctocog Alfa Phase 3 Data Demonstrating Its Potential to Transform the Treatm...

                            Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophili...

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            January 25, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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 : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Major Life Saving Drugs Program Funding for Rare Genetic Disease

                            Details : Cerdelga (eliglustat tartrate) is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs).

                            Product Name : Cerdelga

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 04, 2023

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Immunogen

                            U.S.A
                            German Wound Congress
                            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) : Exagamglogene Autotemcel

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Vertex Pharmaceuticals

                            Deal Size : $352.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            ImmunoGen Announces a Global, Multi-Target License and Option Agreement with Vertex for the Use of ImmunoGen's...

                            Details : Vertex will have the rights to conduct research using ImmunoGen's ADC technology to discover novel targeted conditioning agents for use with gene editing. CTX001 (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edite...

                            Product Name : CTX001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : $15.0 million

                            January 03, 2023

                            Lead Product(s) : Exagamglogene Autotemcel

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Vertex Pharmaceuticals

                            Deal Size : $352.0 million

                            Deal Type : Licensing Agreement

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                            10

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi to present new clinical data reinforcing novel therapies across rare blood disorders at ASH 2022

                            Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            November 30, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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