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Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Hematology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Exagamglogene Autotemcel","moa":"","graph1":"Hematology","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Hematology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Bluebird Bio","pharmaFlowCategory":"DU","therapeuticArea":"Hematology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Lovotibeglogene Autotemcel","moa":"","graph1":"Hematology","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Hematology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Bluebird Bio","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Bluebird Bio"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Nephrology","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Inaxaplin","moa":"","graph1":"Nephrology","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Alpine Immune Sciences","pharmaFlowCategory":"D","therapeuticArea":"Nephrology","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Acquisition","leadProduct":"Povetacicept","moa":"","graph1":"Nephrology","graph2":"Phase I\/ Phase II","graph3":"Vertex Pharmaceuticals","amount2":4.9000000000000004,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Nephrology","amount2New":4.9000000000000004,"dosageForm":"Injection","sponsorNew":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"7","companyTruncated":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Suzetrigine","moa":"","graph1":"Neurology","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"TreeFrog Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Endocrinology","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Licensing Agreement","leadProduct":"VX-880","moa":"","graph1":"Endocrinology","graph2":"Phase I\/ Phase II","graph3":"Vertex Pharmaceuticals","amount2":0.78000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Endocrinology","amount2New":0.78000000000000003,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"7","companyTruncated":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Technology","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Technology","graph2":"Discovery","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Technology","amount2New":0,"dosageForm":"","sponsorNew":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Vanzacaftor","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not 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Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Hematology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Hematology","graph2":"Discovery","graph3":"Vertex Pharmaceuticals","amount2":0.69999999999999996,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Hematology","amount2New":0.69999999999999996,"dosageForm":"","sponsorNew":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Vertex Pharmaceuticals \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Zai Lab","pharmaFlowCategory":"D","therapeuticArea":"Nephrology","country":"U.S.A","productType":"Protein","year":"2025","type":"Collaboration","leadProduct":"Povetacicept","moa":"APRIL\/BAFF","graph1":"Nephrology","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph 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Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Suzetrigine","moa":"NaV1.8","graph1":"Neurology","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"VX-522","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Vertex Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Vertex Pharmaceuticals \/ Inapplicable"}]

Find Clinical Drug Pipeline Developments & Deals by Vertex Pharmaceuticals

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : The agreement aims for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel).

                          Product Name : Casgevy

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          January 30, 2025

                          Lead Product(s) : Exagamglogene Autotemcel

                          Therapeutic Area : Hematology

                          Highest Development Status : Approved FDF

                          Sponsor : NHS England

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          02

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Journavx (suzetrigine) is an investigational oral, selective NaV1.8 inhibitor, which is currently being evaluated for the treatment of adult patients with moderate-to-severe acute pain.

                          Product Name : Journavx

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 30, 2025

                          Lead Product(s) : Suzetrigine

                          Therapeutic Area : Neurology

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Through the collaboration, Zai Lab will be responsible for all development and commercialization activities in the licensed territory once pove (povetacicept) becomes an approved product.

                          Product Name : Pove

                          Product Type : Protein

                          Upfront Cash : Undisclosed

                          January 10, 2025

                          Lead Product(s) : Povetacicept

                          Therapeutic Area : Nephrology

                          Highest Development Status : Phase III

                          Sponsor : Zai Lab

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          04

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Through the collaboration, Vertex will utilize Orna's proprietary LNP delivery solutions to enhance it's efforts in developing next-gen gene editing therapies for patients with SCD and TDT.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : $65.0 million

                          January 07, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Hematology

                          Highest Development Status : Discovery

                          Recipient : Orna Therapeutics

                          Deal Size : $700.0 million

                          Deal Type : Collaboration

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                          05

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Trikafta (ivacaftor/tezacaftor/elexacaftor) is an oral drug designed to binding to different sites on the CFTR protein, indicated for F508del-mutated cystic fibrosis in patients aged 2 years & above.

                          Product Name : Undisclosed

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 20, 2024

                          Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : VX-548 (suzetrigine) is an investigational oral, selective NaV1.8 inhibitor, which is currently being evaluated for the treatment of patients with painful lumbosacral radiculopathy.

                          Product Name : Undisclosed

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 19, 2024

                          Lead Product(s) : Suzetrigine

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : VX-548 (suzetrigine) is an investigational oral, selective NaV1.8 inhibitor, which is currently being evaluated for the treatment of patients with moderate-to-severe acute pain.

                          Product Name : VX-548

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 30, 2024

                          Lead Product(s) : Suzetrigine

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Genomics collaborates with Vertex Pharmaceuticals to use human genetics and machine learning to improve the discovery and development of new precision medicines.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          July 16, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Technology

                          Highest Development Status : Discovery

                          Recipient : Genomics plc

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          09

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : VX-121/VX-661/VX-561 (vanzacaftor/tezacaftor/deutivacaftor) is a combination of three CFTR modulators. It is being evaluated in phase 3 clinial trials for the treatment of Cystic Fibrosis.

                          Product Name : VX-121

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 02, 2024

                          Lead Product(s) : Vanzacaftor,Tezacaftor,Deutivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Kalydeco (ivacaftor) is an oral, CFTR potentiator, small molecule drug candidate. It is now approved for the treatment of cystic fibrosis in patients aged 1 month and older.

                          Product Name : Kalydeco

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 26, 2024

                          Lead Product(s) : Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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