[{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Coave Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"MRT5005","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Bpifrance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Financing","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lysogene \/ Bpifrance","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Bpifrance"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LYS-GM101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lysogene \/ Inapplicable"},{"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-801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"VTX-801","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Vivet Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Vivet Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Bpifrance","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Atamyo Therapeutics \/ Bpifrance","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Bpifrance"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Atamyo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"The Dion Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Partnership","leadProduct":"ATA-200","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atamyo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Atamyo Therapeutics \/ The Dion Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Atamyo Therapeutics \/ The Dion Foundation"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Coave Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Coave Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"CTx-PDE6b","moa":"PDE6B","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Coave Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Coave Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"7","companyTruncated":"Coave Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"GenSight Biologics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Inapplicable"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"GS030","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"GenSight Biologics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GenSight Biologics \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"4BIO Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ 4BIO Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ 4BIO Capital"},{"orgOrder":0,"company":"SparingVision","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Jeito Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Series B Financing","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ Jeito Capital","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Jeito Capital"},{"orgOrder":0,"company":"SparingVision","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Inapplicable"},{"orgOrder":0,"company":"SparingVision","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SparingVision","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"SparingVision \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"SparingVision \/ Inapplicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.

                          Product Name : MRT5005

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          March 26, 2020

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

                          Product Name : MRT5005

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          February 26, 2020

                          Lead Product(s) : MRT5005

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : ATA-200 is a single-injection gene therapy, which is being evaluated for the treatment of the gamma-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).

                          Product Name : ATA-200

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 11, 2024

                          Lead Product(s) : ATA-200

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : VTX-801 is a replication-deficient recombinant adeno-associated viral vector (rAAV) gene therapy, which is being evaluated for the treatment of wilson disease.

                          Product Name : VTX-801

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 04, 2024

                          Lead Product(s) : VTX-801

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : Coave lead program HCTx-PDE6b (AAV2/5-hPDE6B), an AAV5-based gene replacement therapy, is being evaluated in a Phase I/II trial for the treatment of retinitis pigmentosa.

                          Product Name : CTx-PDE6b

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 25, 2024

                          Lead Product(s) : CTx-PDE6b

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : The partnership aims to expand into the US a first-in-human trial of ATA-200, Atamyo’s gene therapy for the treatment of the gamma-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5.

                          Product Name : ATA-200

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 09, 2024

                          Lead Product(s) : ATA-200

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : The Dion Foundation

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          07

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : ATA-200 is a single-injection gene therapy, which is being evaluated for the treatment of the γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).

                          Product Name : ATA-200

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          March 26, 2024

                          Lead Product(s) : ATA-200

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : SPVN06 is a mutation-agnostic AAV gene therapy approach comprising one neurotrophic factor (RdCVF) and one enzyme reducing oxidative stress. It is under development for treating Retinitis Pigmentosa.

                          Product Name : SPVN06

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 24, 2024

                          Lead Product(s) : SPVN06

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : ATA-200, is the gene therapy that delivers a normal copy of the gene for production of γ-sarcoglycan, which is investigated for limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).

                          Product Name : ATA-200

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 19, 2023

                          Lead Product(s) : ATA-200

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Pharmex
                          Not Confirmed
                          Pharmex
                          Not Confirmed

                          Details : SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress used for the treatment of for the treatment of retinitis pigmentosa.

                          Product Name : SPVN06

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 30, 2023

                          Lead Product(s) : SPVN06

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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