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Disorders","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Acquisition","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0.27000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.27000000000000002,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inozyme Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Acquisition","leadProduct":"INZ-701","moa":"Ectonucleotide pyrophosphatase\/phosphodiesterase family member 1 (ENPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0.27000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.27000000000000002,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2024","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2021","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Tyrosine 3-monooxygenase (TH)","graph1":"Musculoskeletal","graph2":"Phase I","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"6","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2023","type":"Inapplicable","leadProduct":"Vosoritide","moa":"Atrial natriuretic peptide receptor B","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin 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Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"CAMP4 Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"3","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Entos Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"}]

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                          01

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : The acquisition will strengthen BioMarin’s enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701, for ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : $270.0 million

                          July 01, 2025

                          Lead Product(s) : INZ-701

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : Inozyme Pharma

                          Deal Size : $270.0 million

                          Deal Type : Acquisition

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                          02

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : The acquisition will strengthen BioMarin's enzyme therapies portfolio, adding a late-stage enzyme replacement therapy, INZ-701, which is currently being assessed for the treatment of ENPP1 Deficiency.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : $270.0 million

                          May 16, 2025

                          Lead Product(s) : INZ-701

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : Inozyme Pharma

                          Deal Size : $270.0 million

                          Deal Type : Acquisition

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                          03

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Through the acquisition, Spruce will focus on the clinical development of AX 250 (tralesinidase alfa) enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Undisclosed

                          April 15, 2025

                          Lead Product(s) : Tralesinidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Spruce Biosciences

                          Deal Size : Undisclosed

                          Deal Type : Acquisition

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                          04

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Palynziq (pegvaliase) is a PEG PAL enzyme that converts phenylalanine to ammonia and trans-cinnamic acid. It reduces blood phenylalanine concentrations in patients with PKU.

                          Product Name : Palynziq

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          April 02, 2025

                          Lead Product(s) : Pegvaliase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          January 17, 2025

                          Lead Product(s) : Pegvaliase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          December 17, 2024

                          Lead Product(s) : BMN 349

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          October 31, 2024

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          June 12, 2024

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy approved for severe hemophilia A, that uses an adeno-associated virus 5, codes for human Factor VIII.

                          Product Name : Roctavian

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 08, 2024

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ChemOutsourcing
                          Not Confirmed
                          ChemOutsourcing
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          April 24, 2024

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Endocrinology

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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