[{"orgOrder":0,"company":"NHS England","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"NHS England","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"NHS England \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"11","companyTruncated":"NHS England \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2020","type":"Not Applicable","leadProduct":"Elexacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2021","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2021","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2021","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2021","type":"Not Applicable","leadProduct":"Elexacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"AIFA","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ AIFA","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ AIFA"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"French Health Authorities for the cystic fibrosis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ French Health Authorities for the cystic fibrosis","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ French Health Authorities for the cystic fibrosis"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Vanzacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","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":"2021","type":"Not Applicable","leadProduct":"Elexacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","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":"2021","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex 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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":"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":"2023","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic 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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 Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","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":"","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||CFTR protein level","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Vertex Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Deutivacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Inapplicable"}]

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                          01

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          Not Confirmed

                          Details : Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), a once-daily next-in-class CFTR modulator, which is indicated for the treatment of cystic fibrosis.

                          Product Name : Alyftrek

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 07, 2025

                          Lead Product(s) : Deutivacaftor,Tezacaftor,Vanzacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) is an oral medicine designed to bind on CFTR protein, which is investigated for cystic fibrosis in patients aged 2 years & above.

                          Product Name : Kaftrio

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 28, 2025

                          Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          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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                          04

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          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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                          05

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          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

                          February 05, 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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                          06

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) is an oral medicine designed to binding to different sites on the CFTR protein, which is investigated for the treatment of cystic fibrosis in patients aged 2 years & above.

                          Product Name : Kaftrio

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 24, 2023

                          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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                          07

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) is an oral medicine designed to binding to different sites on the CFTR protein, which is investigated for the treatment of cystic fibrosis in patients aged 2 years & above.

                          Product Name : Kaftrio

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 23, 2023

                          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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                          08

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          Not Confirmed

                          Details : Trikafta is a combination drug where elexacaftor and tezacaftor bind to different sites of CFTR and additionally facilitates the cellular processing and trafficking of mutated CFTR. Ivacaftor potentiates the channel open probability of the CFTR protein a...

                          Product Name : Trikafta

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 26, 2023

                          Lead Product(s) : Elexacaftor,Tezacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          Not Confirmed

                          Details : TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), is prescription medicine for treatment of cystic fibrosis. Based on analysis through Week 144 of 192-week study, people receiving TRIKAFTA maintained improvement in lung function, respiratory sym...

                          Product Name : Trikafta

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 03, 2022

                          Lead Product(s) : Elexacaftor,Tezacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          European Pharma Congress
                          Not Confirmed
                          European Pharma Congress
                          Not Confirmed

                          Details : TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) real-world safety and effectiveness interim results show improved lung function and significant reductions in risk of pulmonary exacerbations, lung transplant and death for people with cystic fi...

                          Product Name : Trikafta

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 10, 2022

                          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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