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Hoffmann-La Roche \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"AB2 Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Tadekinig Alpha","moa":"IL-18 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AB2 Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"AB2 Bio \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"AB2 Bio \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Inclisiran","moa":"PCSK9 mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Inclisiran","moa":"PCSK9 mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"OAV-101","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"Survival motor neuron protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Undisclosed"}]

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                          01

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : PHVS416 (deucrictibant) is a novel, potent, oral small-molecule bradykinin B2 receptor antagonist currently in development to prevent the occurrence of bradykinin-mediated angioedema attacks.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 01, 2025

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : Zolgensma (onasemnogene abeparvovec) is USFDA approved, one time gne therapy that acts as SMN1 gene stimulant. It is indicated for the treatment of spinal muscular atrophy.

                          Product Name : Zolgensma

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : Onasemnogene Abeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : Zolgensma (onasemnogene abeparvovec) is USFDA approved, one time gne therapy that acts as SMN1 gene stimulant. It is indicated for the treatment of spinal muscular atrophy.

                          Product Name : Zolgensma

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          December 30, 2024

                          Lead Product(s) : Onasemnogene Abeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : Deucrictibant is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Angioedemas, Hereditary.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 08, 2024

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : Deucrictibant is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Angioedemas, Hereditary.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 01, 2024

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : Inclisiran is a Oligonucleotide drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Homozygous Familial Hypercholesterolemia.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          September 19, 2024

                          Lead Product(s) : Inclisiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : Crizanlizumab is a Antibody drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Anemia, Sickle Cell.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          June 03, 2024

                          Lead Product(s) : Crizanlizumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : OCTA-C1-INH is a drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Angioedemas, Hereditary.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          April 12, 2024

                          Lead Product(s) : OCTA-C1-INH

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : Deucrictibant is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Angioedemas, Hereditary.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 03, 2024

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Aus. Peptide Conference
                          Not Confirmed
                          Aus. Peptide Conference
                          Not Confirmed

                          Details : The financing will be used to fund the upcoming Phase 3 studies of ColiFin (colistimethate sodium), an inhaled antibiotic therapeutic for the management of chronic infections in cystic fibrosis patients.

                          Product Name : ColiFin

                          Product Type : Antibiotic

                          Upfront Cash : Undisclosed

                          August 15, 2023

                          Lead Product(s) : Colistimethate Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : SPRIM Global Investments

                          Deal Size : $2.5 million

                          Deal Type : Financing

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