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    [{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"CLN7","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"EcoR1 Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series B Financing","leadProduct":"Undisclosed","moa":"CLN5","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ EcoR1 Capital","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene \/ EcoR1 Capital"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Great Point Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Great Point Partners","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Great Point Partners"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"}]

    Find Clinical Drug Pipeline Developments & Deals by Neurogene

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                            01

                            Neurogene

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                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene Reports Positive Data for NGN-401 in Rett Syndrome Trial

                            Details : NGN-401 is a Cell and Gene therapy drug, administered via intracerebroventricular injection, regulating the MECP2 gene for female pediatric patients with Rett Syndrome.

                            Product Name : NGN-401

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            November 11, 2024

                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Neurogene

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                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene Receives RMAT Designation For Rett Syndrome Gene Therapy NGN-401

                            Details : NGN-401 is a Cell and Gene therapy drug, administered via intracerebroventricular injection, regulating the MECP2 gene for female pediatric patients with Rett Syndrome.

                            Product Name : NGN-401

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            July 08, 2024

                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Neurogene

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                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene Expands and Plans Rapid Patient Enrollment for Rett Syndrome Gene Therapy Trial

                            Details : NGN-401 is an investigational AAV9 gene therapy that delivers the full-length human MECP2 gene. It is being evaluated for the treatment of Rett syndrome.

                            Product Name : NGN-401

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 03, 2024

                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Neurogene

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                            German Wound Congress
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                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene Announces NGN-401 Gene Therapy for Rett Syndrome Selected by FDA Pilot Program

                            Details : NGN-401 is a Cell and Gene therapy drug, administered via intracerebroventricular injection, regulating the MECP2 gene for female pediatric patients with Rett Syndrome.

                            Product Name : NGN-401

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 06, 2024

                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Neurogene

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                            German Wound Congress
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                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients wi...

                            Details : NGN-401 is a Cell and Gene therapy drug, administered via intracerebroventricular injection, regulating the MECP2 gene for female pediatric patients with Rett Syndrome.

                            Product Name : NGN-401

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            November 30, 2023

                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Neurogene

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                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Great Point Partners

                            Deal Size : $95.0 million

                            Deal Type : Private Placement

                            DEALS_DEV arrow-down

                            Neurogene and Neoleukin Announce Definitive Merger Agreement

                            Details : The net proceeds will be used to advance the devlopment of Neurogene's pipeline, including NGN-401, a clinical-stage product for Rett syndrome, which uses novel gene regulation technology for a potential best-in-class profile.

                            Product Name : NGN-401

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            July 18, 2023

                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Great Point Partners

                            Deal Size : $95.0 million

                            Deal Type : Private Placement

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                            07

                            Neurogene

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                            German Wound Congress
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                            Neurogene

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                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene Announces FDA Clearance of IND for NGN-401 Gene Therapy for Children with Rett Syndrome

                            Details : NGN-401 is an adeno-associated virus (AAV) gene therapy investigational product that is the first to deliver the full-length human MECP2 gene, under the control of Neurogene’s EXACT self-contained gene regulation technology.

                            Product Name : NGN-401

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            January 23, 2023

                            Lead Product(s) : NGN-401

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Neurogene

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                            German Wound Congress
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                            Neurogene

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : IND Enabling

                            Sponsor : EcoR1 Capital

                            Deal Size : $115.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Neurogene Closes $115 Million Series B Financing

                            Details : Proceeds will accelerate clinical development of multiple gene therapy programs, novel gene therapy product designs, next generation technology platform and state-of-the-art AAV Vector GMP manufacturing capabilities.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            December 16, 2020

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : IND Enabling

                            Sponsor : EcoR1 Capital

                            Deal Size : $115.0 million

                            Deal Type : Series B Financing

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                            09

                            Neurogene

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                            German Wound Congress
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                            Neurogene

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene Announces FDA Orphan Drug Designation for CLN7 Batten Disease Gene Therapy

                            Details : The U.S FDA has granted Neurogene the Orphan Drug Designation for its adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            November 08, 2020

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Neurogene

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                            German Wound Congress
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                            Neurogene

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Neurogene’s CLN7 Gene Therapy Granted FDA Orphan Drug Designation

                            Details : U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            November 08, 2020

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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