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    [{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Lysogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Lysogene","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Venglustat","moa":"Glycosphingolipid pathway","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Sanofi \/ Inapplicable"}]

    Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development in FRANCE

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                            01

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi Halts Venglustat Clinical Programme in ADPKD

                            Details : The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.

                            Product Name : GZ402671

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 06, 2021

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Lysogene

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                            Lysogene

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                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene to Provide Updates and Topline Results from Phase 2/3 AAVance Gene Therapy Clinical Study and Host We...

                            Details : LYS-SAF302 (olenasufligene relduparvovec) delivers a working copy of the human SGSH gene directly to the brain through intra-cerebral injection. Mutations to the SGSH gene are responsible for the developmental, physical and behavioral symptoms of the dis...

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            November 18, 2022

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Lysogene

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                            Lysogene

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                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Provides Updates and Topline Results from Phase 2/3 AAVance Gene Therapy Clinical Study

                            Details : LYS-SAF302 (olenasufligene relduparvovec) is a novel gene therapy in Phase 2/3 development for the treatment of MPS IIIA, or Sanfilippo syndrome type A. LYS-SAF302 delivers a functional copy of the human SGSH gene directly to brain cells using the AAV ca...

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            November 18, 2022

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Lysogene

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                            Lysogene

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                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Provides Clinical Update on AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Tre...

                            Details : Preliminary results evaluating LYS-SAF302 (olenasufligene relduparvovec) in patients with Sanfilippo syndrome type A who have been followed for at least two years demonstrated improvement, stabilization, or slowing down of decline in cognitive-developmen...

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 18, 2022

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Lysogene

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                            Lysogene

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                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Provides Update on Efficacy, Safety and Timelines of the AAVance phase 2/3 Clinical Trial With Gene T...

                            Details : AAVance trial evaluates, effectiveness of a one-time intracerebral delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase gene LYS-SAF302 (olenasufligene relduparvovec) in children with MPS IIIA.

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 24, 2022

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Lysogene

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                            Lysogene

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                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Provides Update on the AAVance Clinical Trial Evaluating LYS-SAF302 in Patients with MPS IIIA

                            Details : AAVance clinical trial is a global Phase 2/3 gene therapy trial for the treatment of Mucopolysaccharidosis. The company remains committed to the LYS-SAF302 development program and the Sanfilippo patient community.

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            October 26, 2020

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Lysogene

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                            Lysogene

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                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Provides Update on the AAVance Clinical Trial Evaluating LYS-SAF302 in Patients with MPS IIIA

                            Details : Company has announced death of a patient in the AAVance study , a phase 2/3 international clinical study evaluating gene therapy treatment of Mucopolysaccharidosis type IIIA (MPS IIIA, known as Sanfilippo Syndrome type A) .

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            October 15, 2020

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Lysogene

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                            Not Confirmed

                            Lysogene

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                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA

                            Details : Following discussions with the U.S. FDA, a clinical hold was issued for the clinical trial AAVance, a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo s...

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 06, 2020

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Lysogene

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                            Lysogene

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                            • fda
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA

                            Details : The AAVance Phase 2/3 clinical study is designed as an open-label, single-arm, multicenter study of LYS-SAF302 for the treatment of MPS IIIA.

                            Product Name : LYS-SAF302

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 25, 2020

                            Lead Product(s) : Olenasufligene Relduparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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