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    [{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2012","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Coagulation Factor IX (Recombinant)","moa":"factor IX","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Grifols International \/ Undisclosed"},{"orgOrder":0,"company":"Grifols International","sponsor":"Grifols International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Alphanate","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grifols International \/ Grifols International","highestDevelopmentStatusID":"8","companyTruncated":"Grifols International \/ Grifols International"},{"orgOrder":0,"company":"Minoryx Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Leriglitazone","moa":"Peroxisome proliferator-activated receptor gamma","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Minoryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Minoryx Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Minoryx Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"SOM Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"SOM3355","moa":"Synaptic vesicle amine transporter (SLC18A2)","graph1":"Genetic Disease","graph2":"Phase II","graph3":"SOM Biotech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"SOM Biotech \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"SOM Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Medibiofarma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"MBF-015","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Medibiofarma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Medibiofarma \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Medibiofarma \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Medibiofarma

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                            Lead Product(s) : MBF-015

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of MBF-015 in Huntington's Disease Patients

                            Details : MBF-015 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Huntington Disease.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            June 24, 2024

                            Lead Product(s) : MBF-015

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            SOM Biotech

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                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy and Safety on SOM3355 in Huntington's Disease Chorea

                            Details : SOM3355 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Huntington Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 27, 2022

                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            SOM Biotech

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                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            SOM Biotech Granted Orphan Drug Designation by the FDA for SOM3355

                            Details : SOM3355 has potential to be an alternative to current VMAT2 inhibitors with a significantly improved side effect profile for the treatment of chorea in Huntington´s disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 18, 2021

                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            SOM Biotech

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                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            SOM Biotech Presents Positive Phase 2a Data With SOM3355 at the 34thECNP Congress 2021

                            Details : SOM3355, an oral vesicular monoamine transporter 2 (VMAT2) inhibitor for the symptomatic treatment of chorea movements associated with Huntington’s disease (HD).

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 04, 2021

                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Minoryx Therapeutics

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                            Lead Product(s) : Leriglitazone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Femal...

                            Details : MIN-102 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Friedreich Ataxia.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            April 17, 2019

                            Lead Product(s) : Leriglitazone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            SOM Biotech

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                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy and Safety of SOM3355 in Huntington's Disease Chorea

                            Details : SOM3355 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Huntington Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 02, 2018

                            Lead Product(s) : SOM3355

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Grifols International

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                            Lead Product(s) : Alphanate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Grifols International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Alphanate in Immune Tolerance Induction Therapy

                            Details : Alphanate is a Protein drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            March 29, 2017

                            Lead Product(s) : Alphanate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Grifols International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Grifols International

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                            Grifols International

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                            Lead Product(s) : Coagulation Factor IX (Recombinant)

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B

                            Details : Coagulation Factor IX (Recombinant) is a Protein drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Hemophilia B.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            March 27, 2017

                            Lead Product(s) : Coagulation Factor IX (Recombinant)

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Grifols International

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                            Grifols International

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                            Lead Product(s) : Alpha-1 Proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Proce...

                            Details : Alpha-1 Proteinase Inhibitor is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            September 13, 2012

                            Lead Product(s) : Alpha-1 Proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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