Seqens Seqens

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[{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"FSIO","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI\u00ae and SYMDEKO\u00ae for Eligible Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"NHS England","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Expansion of Reimbursement Agreement With NHS England","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Pharmaceuticals Reports Positive Phase 3 Study Results for TRIKAFTA\u00ae in People Ages 12 and Older With Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex\u2019s Kaftrio Approved for Use in Europe and Made Accessible on the NHS","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Cystic Fibrosis Foundation","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","amount":"$650.0 million","upfrontCash":"$575.0 million","newsHeadline":"Royalty Pharma Acquires Additional Royalty Interest from the Cystic Fibrosis Foundation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts Vertex\u2019s Supplemental New Drug Applications for TRIKAFTA\u00ae , SYMDEKO\u00ae and KALYDECO\u00ae for Additional CFTR Mutations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces Positive Phase 3 Study for TRIKAFTA\u00ae in Children Ages 6-11 Years With Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves KALYDECO\u00ae (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With CF as Early as Four Months of Age","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EC Approves Vertex's KALYDECO\u00ae as First and Only CFTR Modulator to Treat Eligible Infants With Cystic Fibrosis as Early as Four Months","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"AIFA","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces New Portfolio Reimbursement Agreement in Italy Including KAFTRIO\u00ae, SYMKEVI\u00ae and ORKAMBI\u00ae and KALYDECO\u00ae","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"French Health Authorities for the cystic fibrosis","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces National Reimbursement Agreement in France for KAFTRIO\u00ae for Eligible Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Spanish government","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Reimbursement Agreement in Spain for KAFTRIO\u00ae (ivacaftor\/tezacaftor\/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis 12 Years and Older With At Least One F508del Mutation in the CFTR Gene","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Receives Australian TGA Approval for TRIKAFTA to Treat People With Cystic Fibrosis Ages 12 Years and Older with F508del Mutation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Receives CHMP Positive Opinion for KAFTRIO in Combination With Ivacaftor for Cystic Fibrosis With At Least One F508del Mutation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Pharmaceuticals Adds Additional Treatment Arm to Ongoing Phase 2 Clinical Studies for Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Gets EC Approval for KAFTRIO in Combo with Ivacaftor to Treat Cystic Fibrosis With At Least One F508del Mutation in the CFTR gene","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Australian Pharmaceutical Benefits Scheme","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Reimbursement Agreement in Australia for TRIKAFTA\u00ae (elexacaftor\/tezacaftor\/ivacaftor and ivacaftor) for Patients With Cystic Fibrosis Ages 12 Years and Older With at Least One F508del Mutation in the CFTR Gene","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces U.S. FDA Approval for TRIKAFTA\u00ae in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Health Canada Accepts for Priority Review Vertex Pharma\u2019s sNDA for PrKalydeco To Treat Cystic Fibrosis in Patients from 4 Months to 18 Years of Age","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex to Present Long-Term Data Demonstrating Significant Benefits of Treatment With CFTR Modulators at North American Cystic Fibrosis Conference (NACFC)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Receives CHMP Positive Opinion for KAFTRIO\u00ae (Ivacaftor\/Tezacaftor\/Elexacaftor) in Combination With Ivacaftor in Children With Cystic Fibrosis Ages 6 Through 11","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Pharmaceuticals Reports Positive Topline Results from Monotherapy Arms of Phase 2 Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves KAFTRIO\u00ae (ivacaftor\/tezacaftor\/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Health Canada Grants Marketing Authorization for TRIKAFTA\u00ae (elexacaftor\/tezacaftor\/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 Through 11 years With At Least One F508del Mutation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces U.S. FDA Approval for ORKAMBI\u00ae (lumacaftor\/ivacaftor) in Children With Cystic Fibrosis Ages 12 to <24 months","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Pharmaceuticals Reports Topline Results from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis (CF) Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex to Present New Data on its Portfolio of Cystic Fibrosis Medicines at the 2022 North American Cystic Fibrosis Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces U.S. FDA Approval for TRIKAFTA\u00ae (elexacaftor\/tezacaftor\/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 2 Through 5 With Certain Mutations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Receives CHMP Positive Opinion for ORKAMBI\u00ae (lumacaftor\/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces U.S. FDA Approval for KALYDECO\u00ae (ivacaftor) to Treat Eligible Infants With CF Ages 1 Month and Older","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis (CF) Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves ORKAMBI (lumacaftor\/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Receives CHMP Positive Opinion for KAFTRIO\u00ae (ivacaftor\/tezacaftor\/elexacaftor) in Combination With Ivacaftor for Children With Cystic Fibrosis Ages 2 Through 5","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces European Medicines Agency Validation for Marketing Authorization Application Extension for KAFTRIO\u00ae in Combination With Ivacaftor to Include People With Cystic Fibrosis and Responsive Rare Mutations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves KAFTRIO\u00ae in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Receives CHMP Positive Opinion for KALYDECO\u00ae for the Treatment of Infants With Cystic Fibrosis Ages 1 Month and Older","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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            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.

            Lead Product(s): Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kalydeco

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 23, 2024

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            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.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 24, 2023

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            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.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 23, 2023

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            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.

            Lead Product(s): Elexacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 15, 2023

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            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 CFTR gene.

            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Orkambi

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 05, 2023

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            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.

            Lead Product(s): Exaluren Sulfate,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: ELX-02

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 14, 2023

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            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.

            Lead Product(s): Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kalydeco

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 03, 2023

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            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 CFTR gene.

            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Orkambi

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 27, 2023

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            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 at the cell surface.

            Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 26, 2023

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            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 symptom and CFTR function.

            Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 03, 2022

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