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III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Attralus \/ Sharmila Dorbala","highestDevelopmentStatusID":"10","companyTruncated":"Attralus \/ Sharmila Dorbala"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Adrian Lacy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Adrian Lacy","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Adrian Lacy"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2016","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Jerry Vockley","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Jerry Vockley"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"BioCryst Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Acquisition","leadProduct":"Navenibart","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Astria Therapeutics","amount2":0.69999999999999996,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.69999999999999996,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ BioCryst Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Astria Therapeutics \/ BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies | Leerink Partners | Morgan Stanley | Cantor","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0.25,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies | Leerink Partners | Morgan Stanley | Cantor","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"Glycine transporter 1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Disc medicine","amount2":0.22,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor","highestDevelopmentStatusID":"10","companyTruncated":"Disc medicine \/ Jefferies | Leerink Partners | Morgan Stanley | Cantor"}]

    Find Drugs for Genetic Disease in Phase III Clinical Development

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                            01

                            Sanofi

                            France
                            Bio-Europe
                            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) : Rilzabrutinib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy & Safety of Rilzabrutinib in Patients with Sickle-cell Disease

                            Details : Rilzabrutinib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of Anemia, Sickle Cell.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 16, 2025

                            Lead Product(s) : Rilzabrutinib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            Bio-Europe
                            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 III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Two Fitusiran Phase 3 Studies Published in The Lancet and The Lancet Haematology Highlight Potential to Addres...

                            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 : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            April 04, 2023

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Immunogen

                            U.S.A
                            AIChE 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) : 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 : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : $15.0 million

                            March 01, 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

                            Immunogen

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                            04

                            Sanofi

                            France
                            Bio-Europe
                            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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            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 : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            January 25, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            Bio-Europe
                            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 III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adoles...

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

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            December 22, 2022

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            06

                            Sanofi

                            France
                            Bio-Europe
                            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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            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 : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            November 30, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            07

                            Sanofi

                            France
                            Bio-Europe
                            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 III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Fitusiran Prophylaxis Reduced Bleeds by 61% in People With Hemophilia a or B, With or Without Inhibitors, Comp...

                            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 : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            July 10, 2022

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            Sanofi

                            France
                            Bio-Europe
                            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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pivotal Data Demonstrate Once-weekly Efanesoctocog Alfa Provides Superior Bleed Protection Compared to Prior F...

                            Details : Investigational once-weekly BIVV001 (efanesoctocog alfa) prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            July 10, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Sanofi

                            France
                            Bio-Europe
                            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 Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Efanesoctocog Alfa Breakthrough Therapy Designation for Hemophilia A

                            Details : BIVV001 (efanesoctocog alfa) is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            June 01, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Sanofi

                            France
                            Bio-Europe
                            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 III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult P...

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

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 15, 2022

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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