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BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Liminal BioSciences \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Inapplicable"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Liminal BioSciences \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Inapplicable"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Liminal BioSciences \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Inapplicable"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Liminal BioSciences \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Inapplicable"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Kedrion","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2021","type":"Divestment","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Liminal BioSciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Liminal BioSciences \/ Kedrion","highestDevelopmentStatusID":"10","companyTruncated":"Liminal BioSciences \/ Kedrion"},{"orgOrder":0,"company":"Evolve Biologics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Other Large Molecule","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":"Intravenous Infusion","sponsorNew":"Evolve Biologics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Evolve Biologics \/ Inapplicable"},{"orgOrder":0,"company":"Medicure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Purinergic receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Medicure \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Inapplicable"},{"orgOrder":0,"company":"Medicure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Pyridoxal Phosphate","moa":"Purinergic receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Medicure \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Inapplicable"}]

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                          01

                          EPS
                          Not Confirmed
                          EPS
                          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 : MC-1

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 23, 2024

                          Lead Product(s) : Pyridoxal Phosphate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          EPS
                          Not Confirmed
                          EPS
                          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 : MC-1

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 23, 2023

                          Lead Product(s) : Pyridoxal Phosphate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          EPS
                          Not Confirmed
                          EPS
                          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 : Other Large Molecule

                          Upfront Cash : Inapplicable

                          September 02, 2021

                          Lead Product(s) : Human Immunoglobulin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          EPS
                          Not Confirmed
                          EPS
                          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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                          05

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : The BLA resubmission for Ryplazim® is supported by data from a Phase 2/3 clinical study which evaluated 15 patients, both pediatric and adults, with congenital plasminogen deficiency over 48 weeks of therapy with Ryplazim®.

                          Product Name : Ryplazim

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          September 21, 2020

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          EPS
                          Not Confirmed
                          EPS
                          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

                          August 11, 2020

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : In a pivotal phase 2/3 clinical trial for the treatment of C-PLGD, all of the patients treated with Ryplazim® achieved at least the targeted increase from baseline in their individual trough plasminogen activity levels through 12 weeks of therapy.

                          Product Name : Ryplazim

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          August 09, 2020

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          EPS
                          Not Confirmed
                          EPS
                          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

                          July 12, 2020

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : Two abstracts related to the Company’s clinical development activities of Ryplazim® for the treatment of Congenital Plasminogen Deficiency will be presented at the 62nd American Society of Hematology Annual Meeting and Exposition scheduled to take pla...

                          Product Name : Ryplazim

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          May 11, 2020

                          Lead Product(s) : Plasminogen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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