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Vertex Pharmaceuticals
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Through the acquisiton, Vertex expand its immunology portfolio by including Alpine’s ALPN-303 (povetacicept), is a highly potent and effective dual antagonist of BAFF and APRIL. Currently it is on track to enter late stage trial in second half of 2024 for the treatment of IgA nephropathy.


Lead Product(s): Povetacicept

Therapeutic Area: Nephrology Product Name: ALPN-303

Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule

Recipient: Alpine Immune Sciences

Deal Size: $4,900.0 million Upfront Cash: $4,900.0 million

Deal Type: Acquisition April 10, 2024

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VX-147 (inaxaplin) is an oral, small-molecule inhibitor of APOL1 channel function, which is being evaluated for the treatment of APOL1-mediated kidney disease.


Lead Product(s): Inaxaplin

Therapeutic Area: Nephrology Product Name: VX-147

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 01, 2024

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VX-407 is a first-in-class small molecule corrector that is designed to inhibit PKD1. It is being evaluated for the treatment of autosomal dominant polycystic kidney disease.


Lead Product(s): VX-407

Therapeutic Area: Genetic Disease Product Name: VX-407

Highest Development Status: IND EnablingProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 21, 2024

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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 IIIProduct 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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Casgevy (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is indicated for patients with Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia.


Lead Product(s): Exagamglogene Autotemcel

Therapeutic Area: Hematology Product Name: Casgevy

Highest Development Status: ApprovedProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 13, 2024

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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.


Lead Product(s): Vanzacaftor,Tezacaftor,Deutivacaftor

Therapeutic Area: Genetic Disease Product Name: VX-121

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 05, 2024

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VX-548 (suzetrigine) is a selective NaV1.8 inhibitor, which is being evaluated in phase 3 clinical trials for the treatment of moderate-to-severe acute pain.


Lead Product(s): Suzetrigine

Therapeutic Area: Neurology Product Name: VX-548

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 30, 2024

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Casgevy (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.


Lead Product(s): Exagamglogene Autotemcel

Therapeutic Area: Genetic Disease Product Name: Casgevy

Highest Development Status: ApprovedProduct Type: Cell and Gene therapy

Recipient: CRISPR Therapeutics

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 16, 2024

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Casgevy (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is indicated for patients with Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia.


Lead Product(s): Exagamglogene Autotemcel

Therapeutic Area: Hematology Product Name: Casgevy

Highest Development Status: ApprovedProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 09, 2024

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Under the agreement, Charles River will manufacture Vertex’s CASGEVY (exagamglogene autotemcel) therapy, the world’s first gene-edited therapy targeting severe sickle cell disease.


Lead Product(s): Exagamglogene Autotemcel

Therapeutic Area: Hematology Product Name: Casgevy

Highest Development Status: ApprovedProduct Type: Cell and Gene therapy

Recipient: Charles River Laboratories, Inc

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration December 18, 2023

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