[{"orgOrder":0,"company":"Encoded Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"ETX101","moa":"","graph1":"Neurology","graph2":"Preclinical","graph3":"Encoded Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Encoded Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Encoded Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Encoded Therapeutics","sponsor":"GV","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Series D Financing","leadProduct":"ETX101","moa":"","graph1":"Neurology","graph2":"Preclinical","graph3":"Encoded Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Encoded Therapeutics \/ GV","highestDevelopmentStatusID":"4","companyTruncated":"Encoded Therapeutics \/ GV"},{"orgOrder":0,"company":"Encoded Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Neurology","graph2":"Preclinical","graph3":"Encoded Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Encoded Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Encoded Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Encoded Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"ETX101","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Encoded Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Encoded Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Encoded Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Encoded Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"ETX101","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Encoded Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Encoded Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Encoded Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Encoded Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ETX101","moa":"SCN1A gene expression","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Encoded Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Encoded Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Encoded Therapeutics \/ Inapplicable"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : ETX101 is an AAV9-mediated gene regulation therapy designed to upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons. It is being evaluated for the treatment of Dravet syndrome.

                          Product Name : ETX101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 13, 2025

                          Lead Product(s) : ETX101

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : ETX101, an AAV9-mediated gene therapy, selectively upregulates SCN1A gene expression in GABAergic neurons. It is being studied for treating SCN1A-positive Dravet syndrome in young children.

                          Product Name : ETX101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 26, 2024

                          Lead Product(s) : ETX101

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : ETX101 is an AAV9-mediated gene therapy designed to upregulate SCN1A expression in GABAergic neurons, currently evaluated in phase 1/2 trials for SCN1A-positive Dravet syndrome.

                          Product Name : ETX101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 06, 2024

                          Lead Product(s) : ETX101

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Multiple adaptable DNA sequence-encoded human genetic elements are modality-agnostic and can be combined to customize expression profiles to optimize therapeutic approaches in and beyond the brain.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 18, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Preclinical

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : ETX101, Encoded's lead asset, was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by FDA for the treatment of SCN1A+ Dravet Syndrome.

                          Product Name : ETX101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 22, 2020

                          Lead Product(s) : ETX101

                          Therapeutic Area : Neurology

                          Highest Development Status : Preclinical

                          Sponsor : GV

                          Deal Size : Undisclosed

                          Deal Type : Series D Financing

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                          06

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : ETX101, a gene therapy developed by Encoded Therapeutics to restore the levels of NaV1.1-alpha specifically in GABAergic neurons, is currently progressing toward clinical development for SCN1A-positive Dravet patients.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 19, 2020

                          Lead Product(s) : ETX101

                          Therapeutic Area : Neurology

                          Highest Development Status : Preclinical

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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