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    [{"orgOrder":0,"company":"Viralgen","sponsor":"Sio Gene Therapies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell and Gene therapy","year":"2020","type":"Partnership","leadProduct":"AXO-AAV-GM1","moa":"Beta-galactosidase (GLB1)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Viralgen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Viralgen \/ Sio Gene Therapies","highestDevelopmentStatusID":"7","companyTruncated":"Viralgen \/ Sio Gene Therapies"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Inapplicable"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Inapplicable"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"SB-007","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SpliceBio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SpliceBio \/ Inapplicable"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Inapplicable"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Inapplicable"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Inapplicable"},{"orgOrder":0,"company":"Grifols International","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Alpha-1 Proteinase Inhibitor","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Grifols International \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Grifols International \/ Inapplicable"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Inapplicable"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Inapplicable"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Columbus Venture Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ Columbus Venture Partners","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ Columbus Venture Partners"},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"EIC Accelerator","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Oligonucleotide","year":"2022","type":"Funding","leadProduct":"ATX-01","moa":"MBNL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ARTHEx Biotech","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"ARTHEx Biotech \/ EIC Accelerator","highestDevelopmentStatusID":"7","companyTruncated":"ARTHEx Biotech \/ EIC Accelerator"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            SpliceBio

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                            SpliceBio

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                            Lead Product(s) : SB-007

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Prelimina...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 24, 2025

                            Lead Product(s) : SB-007

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            SpliceBio

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                            Lead Product(s) : SB-007

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            SpliceBio Doses First Patient in Phase 1/2 ASTRA Trial of SB-007 for Stargardt

                            Details : SB-007 is a protein splicing dual AAV gene therapy that delivers the full-length ABCA4 gene. It is being evaluated for the treatment of stargardt disease.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 17, 2025

                            Lead Product(s) : SB-007

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            ARTHEx Biotech

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                            ARTHEx Biotech

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                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            ARTHEx Doses First Patient in ArthemiR™ Trial for Myotonic Dystrophy

                            Details : ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic which is being evaluated for the treatment of Myotonic Dystrophy Type 1.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            October 18, 2024

                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            ARTHEx Biotech

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                            ARTHEx Biotech

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                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            ARTHEx Biotech Receives FDA Rare Pediatric Designation for ATX-01

                            Details : ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic which is being evaluated for the treatment of Myotonic Dystrophy Type 1.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            July 11, 2024

                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            ARTHEx Biotech

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                            SCI Medicinal Chemistry
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                            ARTHEx Biotech

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                            SCI Medicinal Chemistry
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                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of ATX-01 in Participants With DM1

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 08, 2024

                            Lead Product(s) : ATX-01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Grifols International

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                            SCI Medicinal Chemistry
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                            Grifols International

                            Spain arrow
                            SCI Medicinal Chemistry
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                            Lead Product(s) : Alpha-1 Proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhi...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            January 25, 2021

                            Lead Product(s) : Alpha-1 Proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Viralgen

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                            SCI Medicinal Chemistry
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                            Viralgen

                            Spain arrow
                            SCI Medicinal Chemistry
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                            Lead Product(s) : AXO-AAV-GM1

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Sio Gene Therapies

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Axovant and Viralgen Sign Strategic Gene Therapy Development and Manufacturing Partnership

                            Details : Axovant will have access to manufacturing resources for Axovant’s AAV-based gene therapy programs, AXO-AAV-GM1 for GM1 gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis with sufficient capacity to support ongoing development and eventual commercial...

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 15, 2020

                            Lead Product(s) : AXO-AAV-GM1

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Sio Gene Therapies

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            08

                            Grifols International

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                            SCI Medicinal Chemistry
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                            Grifols International

                            Spain arrow
                            SCI Medicinal Chemistry
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                            Lead Product(s) : Alpha1-proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Grifols International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participa...

                            Details : Undisclosed

                            Product Name : Prolastin-C

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            August 17, 2016

                            Lead Product(s) : Alpha1-proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Grifols International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Grifols International

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                            SCI Medicinal Chemistry
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                            Grifols International

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                            SCI Medicinal Chemistry
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                            • WHO-GMP
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                            Lead Product(s) : Alpha1-proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Grifols International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficienc...

                            Details : Undisclosed

                            Product Name : Prolastin-C

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            August 17, 2016

                            Lead Product(s) : Alpha1-proteinase Inhibitor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Grifols International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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