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I\/II","therapeuticAreaShortName":"Hematology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Graphite Bio \/ Kamau Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Kamau Therapeutics"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Hematology","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Exagamglogene Autotemcel","moa":"","graph1":"Hematology","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Hematology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Bluebird 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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : RLYB331

                          Therapeutic Area : Hematology

                          Study Phase : Preclinical

                          Sponsor : Rallybio

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

                          Details : Rallybio plans to prosecute preclinical activities for KY1066/RLYB331 including CMC, and dose-range finding and toxicity studies, which will then support transition of the asset into clinical development.

                          Brand Name : KY1066

                          Molecule Type : Large molecule

                          Upfront Cash : $3.0 million

                          May 10, 2022

                          Lead Product(s) : RLYB331

                          Therapeutic Area : Hematology

                          Highest Development Status : Preclinical

                          Sponsor : Rallybio

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Deferasirox

                          Therapeutic Area : Hematology

                          Study Phase : Approved

                          Sponsor : Oakrum Pharma, LLC

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : JADENU (deferasirox) tablets for oral use and JADENU® Sprinkle (deferasirox) granules are indicated for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age and older.

                          Brand Name : Jadenu

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 22, 2021

                          Lead Product(s) : Deferasirox

                          Therapeutic Area : Hematology

                          Highest Development Status : Approved

                          Sponsor : Oakrum Pharma, LLC

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Aucta Company Banner

                          03

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : Under the agreement, Optime Care will commercialize three Rigel products, which includes Tavalisse (fostamatinib disodium hexahydrate), indicated for the treatment of thrombocytopenia.

                          Brand Name : Tavalisse

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          October 16, 2024

                          Lead Product(s) : Fostamatinib Disodium Hexahydrate

                          Therapeutic Area : Hematology

                          Highest Development Status : Approved

                          Sponsor : Optime Care

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          04

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : Rytstat (desidustat), first-of-its-kind oral tablet being investigated for the treatment for Sickle Cell Disease, in India.

                          Brand Name : Rytstat

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 14, 2024

                          Lead Product(s) : Desidustat

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase II

                          Sponsor : ICMR

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : The collaboration combines Prime Medicine’s precise, multiplex gene editing capabilities with BMS’s broad expertise in development and commercialization of novel ex vivo T-cell therapies.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          September 30, 2024

                          Lead Product(s) : T-Cell Therapy

                          Therapeutic Area : Hematology

                          Highest Development Status : Preclinical

                          Sponsor : Bristol Myers Squibb

                          Deal Size : $3,610.0 million

                          Deal Type : Collaboration

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                          06

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : Prime Medicine will design optimized Prime Editor reagents for a select number of targets, including reagents that use its Prime Assisted Site-Specific Integrase Gene Editing (PASSIGE™) technology.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $110.0 million

                          September 30, 2024

                          Lead Product(s) : T-Cell-based Therapy

                          Therapeutic Area : Hematology

                          Highest Development Status : Preclinical

                          Sponsor : Bristol Myers Squibb

                          Deal Size : $3,610.0 million

                          Deal Type : Collaboration

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                          07

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : Armlupeg (pegfilgrastim) is a biosimilar of Neulasta, that acts as a G-CSF agonist & it is indicated for the treatment of patients suffering from febrile neutropenia.

                          Brand Name : Armlupeg

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 23, 2024

                          Lead Product(s) : Pegfilgrastim

                          Therapeutic Area : Hematology

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : VLX-1005, a small molecule 12-LOX inhibitor, which is being evaluated for the treatment of heparin induced thrombocytopenia.

                          Brand Name : VLX-1005

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          August 14, 2024

                          Lead Product(s) : VLX-1005

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : The collaboration aims to advance the novel anticoagulant ATI-5923 (tecarfarin), designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices.

                          Brand Name : ATI-5923

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          August 06, 2024

                          Lead Product(s) : Tecarfarin

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase I

                          Sponsor : Abbott Laboratories

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          10

                          SupplySide West 2024
                          Not Confirmed
                          SupplySide West 2024
                          Not Confirmed

                          Details : Fibryga is a fibrinogen concentrate which acts as as fibrinogen replacement therapy. It is approved for the treatment of acquired fibrinogen deficiency (AFD).

                          Brand Name : Fibryga

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 01, 2024

                          Lead Product(s) : Fibrinogen

                          Therapeutic Area : Hematology

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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