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    [{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"FXS01","moa":"DGKk","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"SATT Conectus","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"FXS01","moa":"DGKk","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SATT Conectus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"SATT Conectus \/ Lysogene","highestDevelopmentStatusID":"4","companyTruncated":"SATT Conectus \/ Lysogene"},{"orgOrder":0,"company":"Theranexus","sponsor":"Exelixis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2024","type":"Licensing Agreement","leadProduct":"TX01","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Theranexus \/ Exelixis","highestDevelopmentStatusID":"4","companyTruncated":"Theranexus \/ Exelixis"},{"orgOrder":0,"company":"VECT-HORUS","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Series D Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"VECT-HORUS","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"VECT-HORUS \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"VECT-HORUS \/ Undisclosed"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"ATA-100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"BrainVectis","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Acquisition","leadProduct":"BV-CYP01","moa":"CYP46A1","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"BrainVectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BrainVectis \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"BrainVectis \/ AskBio"},{"orgOrder":0,"company":"Cellectis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"IDUA","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cellectis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Inapplicable"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"VTX-803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"VTX-803","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Tenpoint Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Divestment","leadProduct":"SPVN20","moa":"GIRK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ Tenpoint Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"SparingVision \/ Tenpoint Therapeutics"},{"orgOrder":0,"company":"Cellectis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Cellectis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Inapplicable"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"French Government","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"VTX-806","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ French Government","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ French Government"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"VTX-806","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Theranexus

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                            Theranexus

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                            Lead Product(s) : TX01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Exelixis

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Theranexus and Exeltis License Tx01 for Two Rare Neurological Disorders

                            Details : Under the licensing agreement, Exeltis will acquire exclusive rights to market and distribute Theranexus' proprietary product TX01 for treating Gaucher & Niemann-Pick Disease Type C.

                            Product Name : TX01

                            Product Type : Undisclosed

                            Upfront Cash : $2.1 million

                            December 17, 2024

                            Lead Product(s) : TX01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Exelixis

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            02

                            Vivet Therapeutics

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                            Vivet Therapeutics

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                            Lead Product(s) : VTX-806

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            European Commission Grants Orphan Drug Designation for Vivet's Gene Therapy for CTX

                            Details : VTX-806, an Adeno-associated viral (AAV) vector encoding human CYP27A1 is being investigating for the treatment of Cerebrotendinous Xanthomatosis (CTX).

                            Product Name : VTX-806

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 09, 2024

                            Lead Product(s) : VTX-806

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Vivet Therapeutics

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                            Vivet Therapeutics

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                            Lead Product(s) : VTX-806

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : French Government

                            Deal Size : $5.2 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Vivet Receives EUR 4.9 Million for Gene Therapy Development

                            Details : Vivet will use funds to develop VTX-806, aiming to halt or reverse disease progression in cerebrotendinous xanthomatosis patients.

                            Product Name : VTX-806

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            January 02, 2024

                            Lead Product(s) : VTX-806

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : French Government

                            Deal Size : $5.2 million

                            Deal Type : Funding

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                            04

                            SparingVision

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                            SparingVision

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                            Lead Product(s) : SPVN20

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Tenpoint Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Divestment

                            DEALS_DEV arrow-down

                            SparingVision Sells Ex Vivo GIRK Technology to Tenpoint Therapeutics

                            Details : Through the divestment, Tenpoint has acquired SparingVision's ex vivo GIRK technology. SparingVision will remain focused on in vivo genomic medicines, with SPVN20, a GIRK-based gene therapy candidate.

                            Product Name : SPVN20

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            December 07, 2023

                            Lead Product(s) : SPVN20

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Tenpoint Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Divestment

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                            05

                            Cellectis

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                            Cellectis

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Cellectis Presents Data on Two TALEN®-based Gene Therapy Preclinical Programs for Patients with Sickle Cell D...

                            Details : The pre-clinical data presented at ESGCT further demonstrate our ability to leverage TALEN® gene editing technology to potentially address genetic diseases, namely, sickle cell disease and lysosomal storage diseases.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            November 10, 2022

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Lysogene

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                            Lysogene

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                            Lead Product(s) : FXS01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Publishes Positive Preliminary Preclinical Results in the Treatment of Fragile X Syndrome

                            Details : The publication of a peer-reviewed article for FXS01 in EMBO Molecular Medicine showing that adeno-associated viral (AAV) vector-delivered diacylglycerol kinase (DGKk) achieves long-term rescue of fragile X syndrome (FXS) in a mouse model of disease.

                            Product Name : FXS01

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 21, 2022

                            Lead Product(s) : FXS01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Vivet Therapeutics

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                            Vivet Therapeutics

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                            Lead Product(s) : VTX-803

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Mirum Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Mirum Pharmaceuticals Ink License Agreement for Vivet’s Gene Therapy Programs Targeting Progressive Familial...

                            Details : Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the progr...

                            Product Name : VTX-803

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            December 04, 2021

                            Lead Product(s) : VTX-803

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Mirum Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            08

                            SATT Conectus

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                            SATT Conectus

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                            Lead Product(s) : FXS01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Lysogene Enters into an Exclusive Worldwide License Agreement with SATT Conectus

                            Details : Lysogene will be responsible for the preclinical and clinical development, manufacturing, regulatory activities, and commercialization of the drug candidate, globally. which includes FXS01, a gene therapy candidate for the treatment of Fragile X syndrome...

                            Product Name : FXS01

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            November 06, 2021

                            Lead Product(s) : FXS01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Lysogene

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            09

                            Cellectis

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                            Cellectis

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Cellectis Presents Initial Preclinical Data on Two Novel Gene Therapies for Patients with RAG1 Severe Combined...

                            Details : Data presentation based on a preclinical development of a TALEN® based genome editing in T-cells for the treatment of two product candidates targeting primary immunodeficiencies: RAG1 for Severe Combined Immunodeficiency (SCID) and STAT3 for Hyper IgE s...

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            October 19, 2021

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Atamyo Therapeutics

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                            Atamyo Therapeutics

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                            Lead Product(s) : ATA-100

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100,...

                            Details : First approval of ATA-100, was received from UK MHRA its gene therapy for the treatment of the fukutin-related protein limb-girdle muscular dystrophy Type 2I/R9.

                            Product Name : ATA-100

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            June 12, 2021

                            Lead Product(s) : ATA-100

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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