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    [{"orgOrder":0,"company":"Genethon","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Genethon \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Inapplicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Genethon \/ Sarepta Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Poxel","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"PXL770","moa":"AMPK","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Poxel","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Poxel \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Poxel \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            Genethon

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                            Lead Product(s) : GNT0004

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            First Results Of Gene Therapy GNT0004 for Duchenne Muscular Dystrophy Presented

                            Details : GNT0004 is an AAV8 vector-based gene therapy containing a shortened version of the DMD gene encoding dystrophin, targeting treatment for patients with Duchenne muscular dystrophy.

                            Product Name : GNT0004

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 23, 2024

                            Lead Product(s) : GNT0004

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Atamyo Therapeutics

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                            Atamyo Therapeutics

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                            Lead Product(s) : ATA-200

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Bpifrance

                            Deal Size : $8.6 million

                            Deal Type : Financing

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                            ATA-200, Atamyo Therapeutics’ Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5, Reaches Key M...

                            Details : The financing will be used to support company's clinical trials and manufacturing development programs, including ATA-200 gene therapy targeting γ-sarcoglycan (SGCG) related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).

                            Product Name : ATA-200

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 19, 2023

                            Lead Product(s) : ATA-200

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Bpifrance

                            Deal Size : $8.6 million

                            Deal Type : Financing

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                            03

                            Poxel

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                            Lead Product(s) : PXL770

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Poxel Announces PXL770 Granted Orphan Drug Designation from the U.S. FDA for Autosomal-Dominant Polycystic Kid...

                            Details : PXL770 is a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. AMPK is a central regulator of multiple metabolic pathways leading to the control of lipid metabolism, glucose homeostasis and inflammation.

                            Product Name : PXL770

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 10, 2022

                            Lead Product(s) : PXL770

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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