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    [{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Aro Biotherapeutics","pharmaFlowCategory":"D","amount":"$1,400.0 million","upfrontCash":"Undisclosed","newsHeadline":"Aro Biotherapeutics and Ionis Enter Licensing and Collaboration Agreement Focused on Cell-Specific Delivery of Antisense Oligonucleotides","therapeuticArea":"Oncology","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Small molecule","productStatus":"Undisclosed","date":"January 2020","url1":"","url2":"","graph1":"Oncology","graph2":"Undisclosed"},{"orgOrder":0,"company":"Akcea Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Akcea and Ionis report positive topline Phase 2 results of AKCEA-APOCIII-L Rx","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase II"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. 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Donidalorsen Late-stage Clinical Progress in HAE","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Scipher Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Scipher Medicine Signs Multi-Target Partnership with Ionis","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Discovery Platform"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","amount":"$3,800.0 million","upfrontCash":"$200.0 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Pharmaceuticals","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$60.0 million","newsHeadline":"Ionis Enters Collaboration to Advance Next Generation Program Targeting Lp(a) for Cardiovascular Disease","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$60.0 million","newsHeadline":"Ionis Enters Agreement with Roche for Two Novel RNA-targeted Programs for Alzheimer's Disease and Huntington's Disease","therapeuticArea":"Neurology","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Preclinical"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis Shares Positive Clinical Update from Ongoing Trial of ION582 for Angelman syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$65.0 million","newsHeadline":"Ionis Announces European Licensing Agreement with Otsuka for Donidalorsen in Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis Announces Positive Topline Results from Phase 3 OASIS-HAE Study of Investigational Donidalorsen in Patients with Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis Announces Positive Topline Results from Phase 3 OASIS-HAE Study of Investigational Donidalorsen in Patients with Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Wainua (eplontersen) Granted First-Ever Regulatory Approval in the US for the Treatment of Adults with Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis","therapeuticArea":"Neurology","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Approved"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Olezarsen Receives Orphan Drug designation from U.S. FDA for Familial Chylomicronemia Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eplontersen granted U.S. FDA Fast Track Designation for Patients with Transthyretin-mediated Amyloid Cardiomyopathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis Announces Positive Results from Phase 2 Study of ION224, An Investigational Medicine Demonstrating Clinical Efficacy in the Treatment of NASH\/MASH","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis Presents Positive Results from Phase 3 Balance Study of Olezarsen for Familial Chylomicronemia Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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              Ionis Pharmaceuticals

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              Ionis Presents Positive Results from Phase 3 Balance Study of Olezarsen for Familial Chylomicronemia Syndrome

              Details:

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

              Lead Product(s): Olezarsen

              Therapeutic Area: Genetic Disease Product Name: ISIS 678354

              Highest Development Status: Phase III Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable April 07, 2024

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              Ionis Announces Positive Results from Phase 2 Study of ION224, An Investigational Medicine Demonstrating Clinical Efficacy in the Treatment of NASH/MASH

              Details:

              ION224 is a phase 2 investigational LIgand-Conjugated Antisense (LICA) medicine designed to reduce the production of diacylglycerol acyltransferase 2 (DGAT2) to treat patients with metabolic dysfunction-associated steatohepatitis (MASH).

              Lead Product(s): ION224

              Therapeutic Area: Hepatology (Liver, Pancreatic, Gall Bladder) Product Name: ION224

              Highest Development Status: Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable March 13, 2024

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              Olezarsen Receives Orphan Drug designation from U.S. FDA for Familial Chylomicronemia Syndrome

              Details:

              AKCEA -APOCIII-LRx (olezarsen) is an RNA-targeted investigational ligand conjugated antisense oligonucleotide medicine, that acts as inhibitor of Apolipoprotein C-III. It is being evaluated for the treatment of familial chylomicronemia syndrome.

              Lead Product(s): Olezarsen

              Therapeutic Area: Genetic Disease Product Name: AKCEA -APOCIII-LRx

              Highest Development Status: Phase III Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 15, 2024

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              Eplontersen granted U.S. FDA Fast Track Designation for Patients with Transthyretin-mediated Amyloid Cardiomyopathy

              Details:

              Wainua (eplontersen) is a transthyretin-directed antisense oligonucleotide. It is being developed for the treatment of transthyretin-mediated amyloid cardiomyopathy.

              Lead Product(s): Eplontersen Sodium

              Therapeutic Area: Genetic Disease Product Name: Wainua

              Highest Development Status: Phase III Product Type: Large molecule

              Recipient: AstraZeneca

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 08, 2024

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              Ionis Announces Positive Topline Results from Phase 3 OASIS-HAE Study of Investigational Donidalorsen in Patients with Hereditary Angioedema

              Details:

              IONIS-PKK-LRx (donidalorsen) is an investigational ligand-conjugated antisense (LICA) medicine designed to target the prekallikrein (PKK) pathway. It is under phase 3 clinical development for the treatment of hereditary angioedema.

              Lead Product(s): Donidalorsen

              Therapeutic Area: Genetic Disease Product Name: IONIS-PKK-LRx

              Highest Development Status: Phase III Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 22, 2024

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              Ionis Announces Positive Topline Results from Phase 3 OASIS-HAE Study of Investigational Donidalorsen in Patients with Hereditary Angioedema

              Details:

              IONIS-PKK-LRx (donidalorsen) is an RNA investigational LIgand-Conjugated Antisense (LICA) medicine designed to precisely target and silence the production of prekallikrein (PKK). It is being evaluated in phase 3 clinical trials for the treatment of hereditary angioedema.

              Lead Product(s): Donidalorsen

              Therapeutic Area: Genetic Disease Product Name: IONIS-PKK-LRx

              Highest Development Status: Phase III Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 22, 2024

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              Wainua (eplontersen) Granted First-Ever Regulatory Approval in the US for the Treatment of Adults with Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

              Details:

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

              Lead Product(s): Eplontersen Sodium

              Therapeutic Area: Neurology Product Name: Wainua

              Highest Development Status: Approved Product Type: Large molecule

              Recipient: AstraZeneca

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable December 21, 2023

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              Ionis Announces European Licensing Agreement with Otsuka for Donidalorsen in Hereditary Angioedema

              Details:

              Under the agreement, Otsuka obtains exclusive rights in Europe to commercialize donidalorsen, an investigational LIgand-Conjugated Antisense (LICA) medicine designed to target the prekallikrein, or PKK, pathway, for hereditary angioedema (HAE).

              Lead Product(s): Donidalorsen

              Therapeutic Area: Genetic Disease Product Name: IONIS-PKK-LRx

              Highest Development Status: Phase III Product Type: Large molecule

              Partner/Sponsor/Collaborator: Otsuka Pharmaceutical

              Deal Size: Undisclosed Upfront Cash: $65.0 million

              Deal Type: Licensing Agreement December 18, 2023

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              Ionis Shares Positive Clinical Update from Ongoing Trial of ION582 for Angelman syndrome

              Details:

              ION582 is designed to unsilence the paternal UBE3A allele in order to increase production of the UBE3A protein in the brain, which is investigated for the treatment of Angelman syndrome.

              Lead Product(s): ION582

              Therapeutic Area: Genetic Disease Product Name: ION582

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable November 11, 2023

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              Ionis Enters Agreement with Roche for Two Novel RNA-targeted Programs for Alzheimer's Disease and Huntington's Disease

              Details:

              Roche gains exclusive worldwide rights and will be responsible for clinical development, manufacturing, and commercialization for two undisclosed early-stage programs for RNA-targeting investigational medicines for the treatment of Alzheimer's disease and Huntington's disease.

              Lead Product(s): Undisclosed

              Therapeutic Area: Neurology Product Name: Undisclosed

              Highest Development Status: Preclinical Product Type: Large molecule

              Partner/Sponsor/Collaborator: F. Hoffmann-La Roche

              Deal Size: Undisclosed Upfront Cash: $60.0 million

              Deal Type: Licensing Agreement September 27, 2023

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