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Find Clinical Drug Pipeline Developments & Deals by Vertex Pharmaceuticals

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            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 September 25, 2020

            Details:

            Safety data from a cohort of the Phase 3 ARRIVAL study support treatment with KALYDECO in children ages four to <6 months with eligible mutations.

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            Lead Product(s): CTX001

            Therapeutic Area: Genetic Disease Product Name: CTX001

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Recipient: CRISPR Therapeutics

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 22, 2020

            Details:

            European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).

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            Lead Product(s): LNP based mRNA Therapeutics

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Cell and Gene therapy

            Recipient: Moderna Therapeutics

            Deal Size: $455.0 million Upfront Cash: $75.0 million

            Deal Type: Collaboration September 16, 2020

            Details:

            The three-year research collaboration initially will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator protein to be produced.

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            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            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 September 10, 2020

            Details:

            The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies.

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            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            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 September 01, 2020

            Details:

            U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor).

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            Lead Product(s): Elexacaftor,Tezacaftor,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 August 21, 2020

            Details:

            E.C has granted marketing authorization for KAFTRIO® in a combination regimen with ivacaftor to treat people with cystic fibrosis ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.

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            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Phase IV Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2020

            Details:

            The results of this study demonstrate that the triple combination provides significant additional benefit compared to existing CFTR modulator therapy for F/G and F/RF patients and supports the benefit of the medicine for patients with at least one F508del mutation.

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            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Phase IV Product Type: Small molecule

            Recipient: NHS England

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement June 30, 2020

            Details:

            As part of the agreement with NHS England, Vertex has committed to submit ORKAMBI®, SYMKEVI® and KAFTRIO® to the National Institute for Health and Care Excellence (NICE) within an agreed upon timetable, allowing for a period of real-world data collection on the medicines.

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            Lead Product(s): AAV capsid-gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Recipient: Affinia Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 27, 2020

            Details:

            Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.

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            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: FSIO

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement April 21, 2020

            Details:

            Agreement enables the possibility of rapid patient access to future triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor) once approved in Switzerland .

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