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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":"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":"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":"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":"Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Neoleukin Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Merger","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase 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Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"NGN-101","moa":"Undisclosed","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 \/ 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":"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":"Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Merck & Co","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"NL-201","moa":"IL-2 receptor","graph1":"Oncology","graph2":"Phase I","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Merck & Co","highestDevelopmentStatusID":"6","companyTruncated":"Neurogene \/ Merck & Co"},{"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 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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":"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"}]

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

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 19, 2023

                          Lead Product(s) : NGN-401

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Neoleukin Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          06

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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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                          07

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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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                          08

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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

                          Recipient : Neoleukin Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          09

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 12, 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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                          10

                          World Drug Safety EU
                          Not Confirmed
                          World Drug Safety EU
                          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.

                          Product Name : Undisclosed

                          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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