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Loss","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Tofersen","moa":"SOD1","graph1":"Nutrition and Weight Loss","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Nutrition & Weight Loss","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Expanded Collaboration","leadProduct":"Eplontersen","moa":"ATTR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare 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Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"Olezarsen","moa":"APOC3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Theratechnologies","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Theratechnologies"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. 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Hoffmann-La Roche"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Nutrition and Weight Loss","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"ION363","moa":"Undisclosed","graph1":"Nutrition and Weight Loss","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Nutrition & Weight Loss","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Cardiology\/Vascular Diseases","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Vupanorsen","moa":"ANGPTL3","graph1":"Cardiology\/Vascular 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Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"Prekallikrein","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","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":"Nephrology","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"IONIS-FB-LRx","moa":"Complement factor B","graph1":"Nephrology","graph2":"Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph 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Pharmaceuticals","sponsor":"Praxis Precision Medicines","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"PRAX-222","moa":"SCN2A","graph1":"Neurology","graph2":"IND Enabling","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"5","companyTruncated":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Licensing Agreement","leadProduct":"BIIB115","moa":"SMN protein synthesis","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ionis Pharmaceuticals \/ Biogen","highestDevelopmentStatusID":"4","companyTruncated":"Ionis Pharmaceuticals \/ Biogen"},{"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"}]

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                          01

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          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

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Under the term of license agreement, Theratechnologies receives exclusive rights in Canada for ISIS 678354 (olezarsen), which is being evaluated for FCS and severe hypertriglyceridemia

                          Product Name : ISIS 678354

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          December 04, 2024

                          Lead Product(s) : Olezarsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Theratechnologies

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          03

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : IONIS-PKK-LRx (donidalorsen) is an investigational ligand-conjugated antisense medicine designed to target the prekallikrein pathway. It is under development for treating hereditary angioedema.

                          Product Name : IONIS-PKK-LRx

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          November 04, 2024

                          Lead Product(s) : Donidalorsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : Wainua (eplontersen sodium) is a transthyretin-directed antisense oligonucleotide. It is approved for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis.

                          Product Name : Wainua

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 21, 2024

                          Lead Product(s) : Eplontersen Sodium

                          Therapeutic Area : Neurology

                          Highest Development Status : Approved

                          Recipient : AstraZeneca

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          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 : Large molecule

                          Upfront Cash : Not Applicable

                          October 21, 2024

                          Lead Product(s) : Eplontersen Sodium

                          Therapeutic Area : Neurology

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          Not Confirmed

                          Details : ION373 (zilganersen) is an investigational antisense oligonucleotide medicine being developed as a potential treatment for people with genetically confirmed Alexander disease.

                          Product Name : ION373

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          October 01, 2024

                          Lead Product(s) : Zilganersen

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          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 : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Morgan Stanley

                          Deal Size : $550.3 million

                          Deal Type : Public Offering

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                          08

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          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

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          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 : Large molecule

                          Upfront Cash : $20.0 million

                          June 18, 2024

                          Lead Product(s) : Donidalorsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Otsuka Pharmaceutical

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          10

                          PharmScience R&D
                          Not Confirmed
                          PharmScience R&D
                          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 : Nutrition and Weight Loss

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Biogen

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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