[{"orgOrder":0,"company":"Medexus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"APVO101","moa":"CD19\/CD3","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Medexus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medexus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Medicure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Coagulation factor IIa (F2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medicure \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Undisclosed"},{"orgOrder":0,"company":"Medicure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Coagulation factor IIa (F2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medicure \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Undisclosed"},{"orgOrder":0,"company":"Medicure","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Coagulation factor IIa (F2)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medicure \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Undisclosed"},{"orgOrder":0,"company":"Medexus Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"IB1001","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Medexus Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Medexus Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"CANGENE CORPORATION","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"IB1001","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"CANGENE CORPORATION","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CANGENE CORPORATION \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CANGENE CORPORATION \/ Undisclosed"},{"orgOrder":0,"company":"Evolve Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Human Immunoglobulin","moa":"Fc receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Evolve Biologics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Evolve Biologics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Evolve Biologics \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Undisclosed"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Kedrion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2021","type":"Divestment","leadProduct":"Plasminogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Infusion","sponsorNew":"Liminal BioSciences \/ Kedrion","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Kedrion"},{"orgOrder":0,"company":"Therapure Biomanufacturing","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Therapure Biomanufacturing","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Therapure Biomanufacturing \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Therapure Biomanufacturing \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Alliance Global Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Public Offering","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Alliance Global Partners","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Alliance Global Partners"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Altasciences Company Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Altasciences Company Inc","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Altasciences Company Inc"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Oxypurinol","moa":"Xanthine dehydrogenase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Xortx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Xortx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Undisclosed"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : MC-1 (pyridoxal phosphate) is a naturally occuring purinergic receptor antagonist, which is being evaluated for the treatment of Pyridox(am)Ine 5'-Phosphate Oxidase (PNPO) Deficiency.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 23, 2024

                          Lead Product(s) : Pyridoxal Phosphate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : FDA provides complete approval to enroll patients in its pivotal clinical trial to evaluate "MC-1" (a naturally occurring metabolite of pyridoxine) for treatment of a rare pediatric disease called pyridox(am)ine 5′-phosphate oxidase deficiency.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 23, 2023

                          Lead Product(s) : Pyridoxal Phosphate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : XORLO (proprietary oxypurinol formulation) is in under development for the treatment of individuals with progressing autosomal dominant polycystic kidney disease (ADPKD).

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          August 29, 2023

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : XORLO (proprietary oxypurinol formulation) is in under development for the treatment of individuals with progressing autosomal dominant polycystic kidney disease (ADPKD).

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 04, 2023

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : Following administration of first dose of drug, blood sampling and bioanalytical evaluation will be conducted to characterize pharmacokinetics and bioavailability of XRx-008 novel proprietary formulations of oxypurinol.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 03, 2022

                          Lead Product(s) : Oxypurinol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : Kedrion will enter into an option agreement with Liminal BioSciences to acquire the remainder of the company’s plasma-derived business, as well as Plasminogen business operated through its subsidiaries including Prometic Biotherapeutics which holds the...

                          Product Name : Ryplazim

                          Product Type : Protein

                          Upfront Cash : Undisclosed

                          May 17, 2021

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Kedrion

                          Deal Size : $22.0 million

                          Deal Type : Divestment

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                          07

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : Multicenter study of the efficacy, safety, tolerability, and pharmacokinetics of PlasmaCap IG in adults and children with primary immune deficiency disease met all primary and secondary endpoints.

                          Product Name : PlasmaCap IG

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          February 09, 2021

                          Lead Product(s) : Human Immunoglobulin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : Pyridoxal Phosphate is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Pyridoxamine 5-Prime-Phosphate Oxidase Deficiency.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          January 12, 2021

                          Lead Product(s) : Pyridoxal Phosphate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : Two posters were presented that highlighted individual case histories of patients with C-PLGD treated with the investigational intravenous Ryplazim® (plasminogen) in compassionate use and expanded access cases.

                          Product Name : Ryplazim

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          December 07, 2020

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Neuroscience
                          Not Confirmed
                          Neuroscience
                          Not Confirmed

                          Details : The BLA resubmission for Ryplazim® was supported by data from a Phase 2/3 clinical trial, which evaluated 15 patients, both pediatric and adults, with C-PLGD over 48 weeks of therapy with Ryplazim®.

                          Product Name : Ryplazim

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          November 08, 2020

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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