[{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AAV-based engineered transgene","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AAV-based gene therapy","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AAV-based engineered transgene","moa":"CLN7 gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"EcoR1 Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Series B Financing","leadProduct":"AAV-based gene therapy","moa":"CLN5","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurogene","amount2":0.12,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"","sponsorNew":"Neurogene \/ EcoR1 Capital","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene \/ EcoR1 Capital"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"NGN-401","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Neoleukin Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene 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Partners","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Great Point Partners"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"NGN-401","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Neoleukin Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Merger","leadProduct":"NGN-401","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Neurogene","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Neurogene"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"NGN-401","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"NGN-401","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"NGN-401","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Neurogene \/ Not Applicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"NGN-401","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Neurogene \/ Not Applicable"}]

Find Clinical Drug Pipeline Developments & Deals by Neurogene

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 11, 2024

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          August 07, 2024

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          June 03, 2024

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 04, 2024

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          Details : The combined company will focus on advancing Neurogene’s pipeline of differentiated genetic medicines, including NGN-401, a clinical-stage product for Rett syndrome, which uses novel gene regulation technology for a potential best-in-class profile.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 19, 2023

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Neoleukin Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          06

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 30, 2023

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          Details : The combined company will focus on advancing Neurogene’s pipeline of differentiated genetic medicines, including NGN-401, a clinical-stage product for Rett syndrome, which uses novel gene regulation technology for a potential best-in-class profile.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 18, 2023

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Neoleukin Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          08

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 18, 2023

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Great Point Partners

                          Deal Size : $95.0 million

                          Deal Type : Private Placement

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                          09

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : NGN-401

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          January 23, 2023

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          CPhI India 2024
                          Not Confirmed
                          CPhI India 2024
                          Not Confirmed

                          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.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 16, 2020

                          Lead Product(s) : AAV-based gene therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : EcoR1 Capital

                          Deal Size : $115.0 million

                          Deal Type : Series B Financing

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