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Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Satralizumab","moa":"IL6Ralpha\/GP130","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","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":"Subcutaneous Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","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":"Subcutaneous","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Octapharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Human Normal Immunoglobulin","moa":"High affinity immunoglobulin gamma Fc receptor I; Putative high affinity immunoglobulin gamma Fc receptor IB; Low affinity immunoglobulin gamma Fc region receptor II-a; Low affinity immunoglobulin gamma Fc region receptor II-b; Low affinity immunoglobulin gamma Fc region receptor II-c; Low affinity immunoglobulin gamma Fc region receptor III-A; Low affinity immunoglobulin gamma Fc region receptor III-B; Complement C3; Complement C4-A; Complement C4-B; Complement C5","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Octapharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Octapharma \/ Inapplicable"}]

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                          01

                          ESCRS
                          Not Confirmed
                          ESCRS
                          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 : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 01, 2025

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          ESCRS
                          Not Confirmed
                          ESCRS
                          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 and Gene therapy

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : Onasemnogene Abeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          03

                          ESCRS
                          Not Confirmed
                          ESCRS
                          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 and Gene therapy

                          Upfront Cash : Inapplicable

                          December 30, 2024

                          Lead Product(s) : Onasemnogene Abeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 08, 2024

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 01, 2024

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          June 03, 2024

                          Lead Product(s) : Crizanlizumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ESCRS
                          Not Confirmed
                          ESCRS
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 03, 2024

                          Lead Product(s) : Deucrictibant

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ESCRS
                          Not Confirmed
                          ESCRS
                          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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