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                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Donidalorsen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Approves DAWNZERA As First RNA-Targeted HAE Prophylactic Therapy

                            Details : Dawnzera (donidalorsen sodium) is a prekallikrein-directed antisense oligonucleotide indicated for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older.

                            Product Name : Dawnzera

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            August 21, 2025

                            Lead Product(s) : Donidalorsen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Olezarsen Sodium

                            Therapeutic Area : Cardiology/Vascular Diseases

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ionis Announces Positive Essence Study Results of Olezarsen for Triglycerides

                            Details : Tryngolza (olezarsen) is a RNA-targeted medicine being evaluated for sHTG. It is designed to lower apoC-III production, a liver produced protein that regulates triglyceride metabolism in blood.

                            Product Name : Tryngolza

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            May 19, 2025

                            Lead Product(s) : Olezarsen Sodium

                            Therapeutic Area : Cardiology/Vascular Diseases

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ION582

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

                            Details : ION582 is a Oligonucleotide drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Angelman Syndrome.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            April 06, 2025

                            Lead Product(s) : ION582

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : BIIB080

                            Therapeutic Area : Neurology

                            Study Phase : Phase II

                            Recipient : Biogen

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biogen’s BIIB080 Gets FDA Fast Track for Tau-Targeting Alzheimer’s Therapy

                            Details : BIIB080 is an investigational antisense oligonucleotide (ASO) therapy designed to target microtubule-associated protein tau (MAPT) mRNA to reduce the production of tau protein.

                            Product Name : IONIS-MAPTRx

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            April 02, 2025

                            Lead Product(s) : BIIB080

                            Therapeutic Area : Neurology

                            Highest Development Status : Phase II

                            Recipient : Biogen

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : Olezarsen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Ionis, Sobi Expand Partnership for Olezarsen Commercialization Outside U.S.

                            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 : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            06

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : Sapablursen

                            Therapeutic Area : Oncology

                            Study Phase : Phase II

                            Sponsor : Ono Pharmaceutical

                            Deal Size : $940.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Ionis and Ono Announce Global License Agreement for Sapablursen in Polycythemia Vera

                            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 : Undisclosed

                            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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                            07

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Eplontersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            WAINZUA Approved in EU for Hereditary Transthyretin Amyloidosis

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Olezarsen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            US FDA Approves Ionis Pharma's Genetic Disorder Drug

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Olezarsen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Theratechnologies

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Theratechnologies Licenses Olezarsen and Donidalorsen from Ionis for Canada

                            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 : Undisclosed

                            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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                            10

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Organic Process R&D
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : Donidalorsen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ionis Announces FDA Review of NDA for Donidalorsen in HAE Prophylaxis

                            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 : Oligonucleotide

                            Upfront Cash : Inapplicable

                            November 04, 2024

                            Lead Product(s) : Donidalorsen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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