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Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Donidalorsen","moa":"Prekallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Agreement","leadProduct":"Nusinersen Sodium","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Ionis Pharmaceuticals","amount2":1.1299999999999999,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":1.1299999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Royalty Pharma","highestDevelopmentStatusID":"12","companyTruncated":"Ionis Pharmaceuticals \/ Royalty Pharma"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Bepirovirsen","moa":"","graph1":"Infections and Infectious Diseases","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Eplontersen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Tofersen","moa":"","graph1":"Neurology","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Eplontersen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Chinook Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Nephrology","country":"","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"Antisense Oligonucleotide-based Therapy","moa":"","graph1":"Nephrology","graph2":"Discovery","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Chinook Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Ionis Pharmaceuticals \/ Chinook Therapeutics"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Donidalorsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Scipher Medicine","pharmaFlowCategory":"D","therapeuticArea":"Cardiology\/Vascular Diseases","country":"","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"Oligonucleotide Therapy","moa":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Discovery Platform","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"3","companyTruncated":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Cardiology\/Vascular Diseases","country":"","productType":"Large molecule","year":"2023","type":"Expanded Collaboration","leadProduct":"Eplontersen","moa":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":3.7999999999999998,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":3.7999999999999998,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Eplontersen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Cardiology\/Vascular Diseases","country":"","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"Pelacarsen","moa":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ionis Pharmaceuticals \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Neurology","graph2":"Preclinical","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"4","companyTruncated":"Ionis Pharmaceuticals \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ION582","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Licensing Agreement","leadProduct":"Donidalorsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Donidalorsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Donidalorsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Eplontersen Sodium","moa":"","graph1":"Neurology","graph2":"Approved","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Olezarsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis 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Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Hepatology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Olezarsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Biogen","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"ION541","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Biogen","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Biogen"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Otsuka Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Donidalorsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Otsuka Pharmaceutical"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"ION582","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Olezarsen","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"Zilganersen","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Ionis Pharmaceuticals","amount2":0.55000000000000004,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Morgan Stanley","highestDevelopmentStatusID":"14","companyTruncated":"Ionis Pharmaceuticals \/ Morgan Stanley"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Eplontersen Sodium","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"14","companyTruncated":"Ionis Pharmaceuticals \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not 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Pharmaceuticals \/ Not Applicable"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : Wainua

                          Molecule 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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                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

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

                          Brand Name : Wainzua

                          Molecule 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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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

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

                          Brand Name : ION373

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          October 01, 2024

                          Lead Product(s) : Zilganersen

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : ION373

                          Molecule Type : Large molecule

                          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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                          05

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

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

                          Brand Name : ISIS 678354

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 25, 2024

                          Lead Product(s) : Olezarsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : IONIS-PKK-LRx

                          Molecule 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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                          07

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : BIIB105

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 16, 2024

                          Lead Product(s) : ION541

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Biogen

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : ION582

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 16, 2024

                          Lead Product(s) : ION582

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

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

                          Brand Name : ISIS 678354

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          April 07, 2024

                          Lead Product(s) : Olezarsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : ION224 is a phase 2 investigational LICA medicine reducing diacylglycerol acyltransferase 2 production, being evaluated for treating metabolic dysfunction-associated steatohepatitis (MASH).

                          Brand Name : ION224

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 13, 2024

                          Lead Product(s) : ION224

                          Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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