[{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"FSIO","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Lumacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vertex Pharmaceuticals \/ FSIO","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ FSIO"},{"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 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Disease","country":"","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":"","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":"","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":"","productType":"Small molecule","year":"2020","type":"Not 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Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Eukaroyotic ribosomal selective glycoside","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","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":"","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":"","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":"","productType":"Small 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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":"","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 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Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"}]

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                          01

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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.

                          Brand Name : Kalydeco

                          Molecule 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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                          02

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

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

                          Brand Name : Kalydeco

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 23, 2024

                          Lead Product(s) : Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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.

                          Brand Name : Kaftrio

                          Molecule 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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                          04

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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.

                          Brand Name : Kaftrio

                          Molecule 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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                          05

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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 children With cystic fibrosis ages 2 through 5.

                          Brand Name : Kaftrio

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 15, 2023

                          Lead Product(s) : Elexacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Orkambi is a combination of lumacaftor which improves the conformational stability of F508del-CFTR and ivacaftor, a CFTR potentiator. It is being used for cystic fibrosis in patients aged 1 year and older who have two copies of the F508del mutation in CF...

                          Brand Name : Orkambi

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 05, 2023

                          Lead Product(s) : Lumacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : ELX-02 exaluren) is a small molecule drug candidate designed to restore production of full-length functional proteins. It is being developed for the treatment of CF patients with nonsense mutations.

                          Brand Name : ELX-02

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 14, 2023

                          Lead Product(s) : Exaluren Sulfate,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Kalydeco (ivacaftor) is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. it has received USFDA approval to treat eligible infants with CF ages 1 month and older.

                          Brand Name : Kalydeco

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          May 03, 2023

                          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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                          09

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Orkambi is a combination of lumacaftor which improves the conformational stability of F508del-CFTR and ivacaftor is a CFTR potentiator. It is being used for cystic fibrosis in patients aged 1 year and older who have two copies of the F508del mutation in ...

                          Brand Name : Orkambi

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 27, 2023

                          Lead Product(s) : Lumacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          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...

                          Brand Name : Trikafta

                          Molecule 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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