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Vafseo (vadadustat) is a once-daily oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor. It is approved for the treatment of anemia due to chronic kidney disease in adult patients on dialysis.


Lead Product(s): Vadadustat

Therapeutic Area: Nephrology Product Name: Vafseo

Highest Development Status: ApprovedProduct Type: Small molecule

Recipient: Akebia Therapeutics

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 27, 2024

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Ferinject (ferric carboxymaltose) is an Fe modulator, small molecule drug candidate. It is approved for the treatment of iron deficiency anemia in adult and pediatric patients one year of age and older when oral iron preparations are not tolerated.


Lead Product(s): Ferric Carboxymaltose

Therapeutic Area: Hematology Product Name: Ferinject

Highest Development Status: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 19, 2024

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Filspari (sparsentan) is a once-daily oral medication, designed to selectively target two critical pathways in the disease progression of IgAN (endothelin-1 and angiotensin II). It is approved for the treatment of IgA Nephropathy.


Lead Product(s): Sparsentan

Therapeutic Area: Nephrology Product Name: Filspari

Highest Development Status: ApprovedProduct Type: Small molecule

Recipient: Travere Therapeutics

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 23, 2024

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  • Development Update

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CSL112 (apolipoprotein A-I [human]) is an investigational cholesterol efflux enhancer, developed using a novel formulation of human plasma-derived apoA-I. It is being developed for the treatment of acute myocardial infarction.


Lead Product(s): Apolipoprotein A-I, Human

Therapeutic Area: Cardiology/Vascular Diseases Product Name: CSL112

Highest Development Status: Phase IIIProduct Type:

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 11, 2024

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  • Development Update

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Hemgenix (etranacogene dezaparvovec-drlb) is an AAV5 based gene therapy designed to deliver a copy of a gene encoding hFIX-Padua. Its single IV infusion results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.


Lead Product(s): Etranacogene Dezaparvovec

Therapeutic Area: Genetic Disease Product Name: Hemgenix

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 15, 2024

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Zemaira (human alpha1-proteinase inhibitor) is a highly purified form of AAT (human) currently approved for chronic augmentation and maintenance therapy in adults with Alpha 1 Antitrypsin Deficiency and clinical evidence of emphysema.


Lead Product(s): Human Alpha1-Proteinase Inhibitor

Therapeutic Area: Genetic Disease Product Name: Zemaira

Highest Development Status: ApprovedProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 02, 2024

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  • Development Update

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CSL312 (garadacimab) is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb) that has completed Phase 3 clinical development as a potential new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE.


Lead Product(s): Garadacimab

Therapeutic Area: Genetic Disease Product Name: CSL312

Highest Development Status: Phase IIIProduct Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 14, 2023

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  • Development Update

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ARCT-154 is an self-amplifying mRNA (sa-mRNA) vaccine based on the SARS-CoV-2 D614G variant spike protein. This covid-19 vaccine for initial vaccination and booster for adults 18 years and older.


Lead Product(s): ARCT-154

Therapeutic Area: Infections and Infectious Diseases Product Name: LUNAR-COV19

Highest Development Status: ApprovedProduct Type: Vaccine

Recipient: Arcturus Therapeutics

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 28, 2023

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  • Development Update

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Hemgenix (etranacogene dezaparvovec-drlb) is an AAV5 based gene therapy designed to deliver a copy of a gene encoding hFIX-Padua. Its single IV infusion results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.


Lead Product(s): Etranacogene Dezaparvovec-drlb

Therapeutic Area: Genetic Disease Product Name: Hemgenix

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 October 26, 2023

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  • Deals

Details:

These Global RAI awardees’ research addresses important unmet medical needs in several of CSL’s therapeutic areas, which include immunology, nephrology and transplant, respiratory, hematology, cardiovascular and metabolic disease, and vaccines.


Lead Product(s): Undisclosed

Therapeutic Area: Cardiology/Vascular Diseases Product Name: Undisclosed

Highest Development Status: DiscoveryProduct Type: Undisclosed

Recipient: Institute of Cardiovascular and Metabolic Diseases

Deal Size: $0.3 million Upfront Cash: Undisclosed

Deal Type: Funding October 04, 2023

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