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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":"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":"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":"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":"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":"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":"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":"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":"Injection","sponsorNew":"Lysogene \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Lysogene \/ Inapplicable"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Ultevursen","moa":"usherin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Laboratoires Thea","sponsor":"Sepul Bio","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Ultevursen","moa":"usherin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Laboratoires Thea","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Laboratoires Thea \/ Sepul Bio","highestDevelopmentStatusID":"9","companyTruncated":"Laboratoires Thea \/ Sepul Bio"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Venglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"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

                            France
                            CPhI WW Frankfurt
                            Attending
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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 : Undisclosed

                            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

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
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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

                            A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 14, 2018

                            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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                            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 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 : Undisclosed

                            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 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 : Undisclosed

                            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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                            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 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 : Undisclosed

                            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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                            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 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 : Undisclosed

                            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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                            07

                            Laboratoires Thea

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                            Laboratoires Thea

                            France arrow
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                            Not Confirmed
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                            Lead Product(s) : QR-421a

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Sepul Bio

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            January 04, 2022

                            Lead Product(s) : QR-421a

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Sepul Bio

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Laboratoires Thea

                            France arrow
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                            Not Confirmed

                            Laboratoires Thea

                            France arrow
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                            Lead Product(s) : Ultevursen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Sepul Bio

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in E...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            December 15, 2021

                            Lead Product(s) : Ultevursen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Sepul Bio

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Lysogene

                            France arrow
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                            Not Confirmed

                            Lysogene

                            France arrow
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                            Not Confirmed
                            • fda
                            • EDQM
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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 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 : Undisclosed

                            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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                            10

                            Lysogene

                            France arrow
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                            Lysogene

                            France arrow
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                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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 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 : Undisclosed

                            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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