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    [{"orgOrder":0,"company":"Genethon","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GNT-0003","moa":"UGT1A1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Genethon \/ Hansa Biopharma","highestDevelopmentStatusID":"8","companyTruncated":"Genethon \/ Hansa Biopharma"},{"orgOrder":0,"company":"Genethon","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GNT-003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Genethon \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Inapplicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Genethon \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Inapplicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Genethon \/ Sarepta Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Genethon","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Imlifidase","moa":"Antibodies","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lyophilized Powder","sponsorNew":"Genethon \/ Hansa Biopharma","highestDevelopmentStatusID":"8","companyTruncated":"Genethon \/ Hansa Biopharma"},{"orgOrder":0,"company":"Genethon","sponsor":"Genethon","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Divalproex Sodium","moa":"Succinate semialdehyde dehydrogenase","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genethon \/ Genethon","highestDevelopmentStatusID":"8","companyTruncated":"Genethon \/ Genethon"}]

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                            01

                            Genethon

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                            Lead Product(s) : GNT-0003

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Hansa Biopharma

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Genethon/Hansa Start Phase 2 Trial Of Imlifidase Pre-Treatment for Crigler-Najjar

                            Details : GNT-0003 is being developed for crigler-najjar syndrome. The drug candidate is administered through intravenous route. Direct administration of an AAV vector carrying a normal copy of UGT1A1.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            December 03, 2024

                            Lead Product(s) : GNT-0003

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Hansa Biopharma

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Genethon

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                            Lead Product(s) : Imlifidase

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Hansa Biopharma

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            July 24, 2024

                            Lead Product(s) : Imlifidase

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Hansa Biopharma

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Genethon

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                            Lead Product(s) : GNT0004

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            First Results Of Gene Therapy GNT0004 for Duchenne Muscular Dystrophy Presented

                            Details : GNT0004 is an AAV8 vector-based gene therapy containing a shortened version of the DMD gene encoding dystrophin, targeting treatment for patients with Duchenne muscular dystrophy.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 23, 2024

                            Lead Product(s) : GNT0004

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Genethon

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                            Genethon

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                            Lead Product(s) : GNT-003

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

                            Details : GNT-0003 is being developing a gene therapy treatment to treat patients with severe forms of CN syndrome by restoring expression of the UGT1A1 enzyme in the patient’s liver.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            October 01, 2023

                            Lead Product(s) : GNT-003

                            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

                            Genethon

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                            Genethon

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                            Lead Product(s) : Divalproex Sodium

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Recipient : Centre d'Etude des Cellules Souches

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            June 25, 2021

                            Lead Product(s) : Divalproex Sodium

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Recipient : Centre d'Etude des Cellules Souches

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Genethon

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                            Lead Product(s) : GNT0004

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Sarepta Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy produ...

                            Details : CDMO’s capacity to produce AAV materials at large-scale will support Genethon and Sarepta’s project to take micro-dystrophin to clinical trials this year.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            September 01, 2020

                            Lead Product(s) : GNT0004

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Sarepta Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            07

                            Genethon

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                            Lead Product(s) : GNT0003

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Undisclosed

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Gene Therapy for Severe Crigler Najjar Syndrome

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            March 15, 2018

                            Lead Product(s) : GNT0003

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Undisclosed

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Genethon

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                            Genethon

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                            Lead Product(s) : X Vivo Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Gene Therapy for X-linked Chronic Granulomatous Disease

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            May 02, 2016

                            Lead Product(s) : X Vivo Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Genethon

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                            Genethon

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                            Lead Product(s) : CD34+ Cell Transduced with WASP Lentiviral Vector

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            January 07, 2015

                            Lead Product(s) : CD34+ Cell Transduced with WASP Lentiviral Vector

                            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

                            Genethon

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                            Lead Product(s) : X Vivo Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            May 16, 2013

                            Lead Product(s) : X Vivo Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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