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    [{"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":"Infusion","sponsorNew":"Evolve Biologics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Evolve Biologics \/ Inapplicable"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Inapplicable","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 \/ 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":"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 \/ 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":"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 \/ 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":"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 \/ 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":"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 \/ 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":"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":"Medicure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ 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":"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 \/ 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":"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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Alliance Global Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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 \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Xortx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Altasciences Company Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","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"}]

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                            01

                            Medicure

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                            Lead Product(s) : Pyridoxal Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Medicure Receives US FDA Fast Track Designation for MC-1 for PNPO Deficiency

                            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 : 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

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                            02

                            Medicure

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                            Lead Product(s) : Pyridoxal Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Medicure Announces FDA Provides Complete Approval to Enroll Patients in Its Pivotal Phase 3 Trial for Treatmen...

                            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 : 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

                            Xortx Therapeutics

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                            Lead Product(s) : Oxypurinol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            XORTX Submits Orphan Drug Designation Application to the European Medicines Agency (EMA) to Treat Progressive ...

                            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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 29, 2023

                            Lead Product(s) : Oxypurinol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

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                            04

                            Xortx Therapeutics

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                            Lead Product(s) : Oxypurinol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Confirms Eligibility of XORLO™ for Accelerated Approval

                            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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 05, 2023

                            Lead Product(s) : Oxypurinol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

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                            05

                            Xortx Therapeutics

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                            Lead Product(s) : Oxypurinol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            XORTX Initiates Dosing of Human Subjects in the XRX-OXY-101 Clinical Trial

                            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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 05, 2022

                            Lead Product(s) : Oxypurinol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

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                            06

                            Evolve Biologics

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                            Lead Product(s) : Human Immunoglobulin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Evolve Biologics® Completes Phase III Adult and Pediatric Trials for Plasmacap™ Ig (Intravenous Immunoglobu...

                            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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                            07

                            Liminal BioSciences

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                            Lead Product(s) : Plasminogen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Kedrion

                            Deal Size : $22.0 million

                            Deal Type : Divestment

                            DEALS_DEV arrow-down

                            Liminal BioSciences Announces Closing of Divestiture of Plasma Collection Centers and Enters into Option Agree...

                            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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                            08

                            Medicure

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                            Lead Product(s) : Pyridoxal Phosphate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 12, 2021

                            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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                            09

                            Liminal BioSciences

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                            Liminal BioSciences

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                            Lead Product(s) : Plasminogen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Liminal BioSciences Provides Update on Progress on BLA for Ryplazim®(plasminogen)

                            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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                            10

                            Liminal BioSciences

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                            Liminal BioSciences

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                            Lead Product(s) : Plasminogen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Liminal BioSciences Announces FDA Extension of Review Period for Ryplazim®(Plasminogen) BLA

                            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

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