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Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Olezarsen","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Olezarsen","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Theratechnologies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"Olezarsen","moa":"APOC-3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Theratechnologies","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Theratechnologies"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"Olezarsen Sodium","moa":"APOC-3","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Swedish Orphan Biovitrum AB","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Cardiology\/Vascular Diseases","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Olezarsen","moa":"APOC-3","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Cardiology\/Vascular Diseases","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Vupanorsen","moa":"ANGPTL3","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase II","graph3":"Akcea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Akcea Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Akcea Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Cardiology\/Vascular Diseases","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AKCEA-APOCIII-LRx","moa":"APOC-3","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase II","graph3":"Akcea Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Akcea Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Akcea Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Cardiology\/Vascular Diseases","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AKCEA-APOCIII-LRx","moa":"APOC-3","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase 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Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Nephrology","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fesomersen","moa":"Factor XI","graph1":"Nephrology","graph2":"Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"Ionis Pharmaceuticals \/ Bayer AG"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Nephrology","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fesomersen","moa":"Factor XI","graph1":"Nephrology","graph2":"Phase II","graph3":"Ionis 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Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","country":"U.S.A","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"ION224","moa":"Undisclosed","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Hepatology","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Licensing Agreement","leadProduct":"ION455","moa":"PNPLA3","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Undisclosed","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Hepatology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"1","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Ono Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Oncology","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"Sapablursen","moa":"Matriptase-2","graph1":"Oncology","graph2":"Phase II","graph3":"Ionis Pharmaceuticals","amount2":0.93999999999999995,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Oncology","amount2New":0.93999999999999995,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Ono Pharmaceutical","highestDevelopmentStatusID":"8","companyTruncated":"Ionis Pharmaceuticals \/ Ono Pharmaceutical"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Chinook Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Nephrology","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Nephrology","graph2":"Discovery","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"Oral","sponsorNew":"Ionis Pharmaceuticals \/ Chinook Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Ionis Pharmaceuticals \/ Chinook Therapeutics"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Agreement","leadProduct":"Eplontersen","moa":"ATTR protein","graph1":"Neurology","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Agreement","leadProduct":"Eplontersen","moa":"ATTR protein","graph1":"Neurology","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph 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Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"}]

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                          01

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : Under the licensing agreement, Sobi will hold the exclusive rights in countries outside the U.S., Canada to commercialize Tryngolza (olezarsen sodium) as a potential treatment for FCS.

                          Product Name : Tryngolza

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          March 26, 2025

                          Lead Product(s) : Olezarsen Sodium

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          02

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : Under the licensing agreement, Ono obtains exclusive rights for the development and commercialization of ISIS 702843 (sapablursen), an investigational RNA-targeted medicine for polycythemia vera.

                          Product Name : ISIS 702843

                          Product Type : Oligonucleotide

                          Upfront Cash : $280.0 million

                          March 11, 2025

                          Lead Product(s) : Sapablursen

                          Therapeutic Area : Oncology

                          Highest Development Status : Phase II

                          Sponsor : Ono Pharmaceutical

                          Deal Size : $940.0 million

                          Deal Type : Licensing Agreement

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                          03

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : Waiznua (eplontersen) is a transthyretin-directed antisense oligonucleotide. It is indicated for adults with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.

                          Product Name : Wainzua

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          March 10, 2025

                          Lead Product(s) : Eplontersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : Tryngolza (olezarsen) is a APOC3 inhibitor, antisense oligonucleotide, which is indicated for the treatment of familial chylomicronemia syndrome.

                          Product Name : Tryngolza

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : Olezarsen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : Waiznua (eplontersen) is a transthyretin-directed antisense oligonucleotide. It is indicated for adults with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.

                          Product Name : Wainzua

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          October 21, 2024

                          Lead Product(s) : Eplontersen Sodium

                          Therapeutic Area : Neurology

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : The net proceeds will used to fund the commercial launch company's lead product of ION373 (zilganersen), which is a late-stage clinical program to treat Alexander's disease.

                          Product Name : ION373

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          September 09, 2024

                          Lead Product(s) : Zilganersen

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase III

                          Sponsor : Morgan Stanley

                          Deal Size : $550.3 million

                          Deal Type : Public Offering

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                          07

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : ISIS 678354 (olezarsen) is a APOC3 inhibitor, antisense oligonucleotide, which is being evaluated for the treatment of familial chylomicronemia syndrome.

                          Product Name : ISIS 678354

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 04, 2024

                          Lead Product(s) : Olezarsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : ISIS 678354 (olezarsen) is a APOC3 inhibitor, antisense oligonucleotide, which is being evaluated for the treatment of familial chylomicronemia syndrome.

                          Product Name : ISIS 678354

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          June 25, 2024

                          Lead Product(s) : Olezarsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : Ionis entered into a license agreement with Otsuka & obtained exclusive rights across the Asia-Pacific region for IONIS-PKK-LRx (donidalorsen) for treating hereditary angioedema.

                          Product Name : IONIS-PKK-LRx

                          Product Type : Oligonucleotide

                          Upfront Cash : $20.0 million

                          June 18, 2024

                          Lead Product(s) : Donidalorsen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Otsuka Pharmaceutical

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          10

                          Cophex
                          Not Confirmed
                          Cophex
                          Not Confirmed

                          Details : BIIB105 (ION541) is an investigational oligonucleotide designed to reduce the production of the ataxin-2 (ATXN2) protein. It is being evaluated for the treatment of amyotrophic lateral sclerosis.

                          Product Name : BIIB105

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          May 16, 2024

                          Lead Product(s) : ION541

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Biogen

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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