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    [{"orgOrder":0,"company":"Grace Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"AAV9 Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Grace Science \/ Not Applicable","highestDevelopmentStatusID":"1","companyTruncated":"Grace Science \/ Not Applicable"},{"orgOrder":0,"company":"Grace Science","sponsor":"Andelyn Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Partnership","leadProduct":"GS-100","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Grace Science","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Grace Science"},{"orgOrder":0,"company":"Grace Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"GS-100","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Grace Science \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Not Applicable"},{"orgOrder":0,"company":"Grace Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"GS-100","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Grace Science","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Grace Science \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Grace Science \/ Not Applicable"}]

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                            Lead Product(s) : GS-100

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Grace Science Selected by FDA for START Pilot Program for GS-100 Gene Therapy

                            Details : GS-100 is a recombinant, single-stranded AAV9 vector that encodes a full-length version of the human NGLY1 gene. It is being evaluated for the treatment of NGLY1 Deficiency.

                            Brand Name : GS-100

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            June 03, 2024

                            Lead Product(s) : GS-100

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            02

                            Grace Science

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                            Lead Product(s) : GS-100

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Recipient : Andelyn Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            Andelyn Biosciences Partners for AAV Gene Therapy Manufacturing for NGLY1 Deficiency

                            Details : The partnership aims to tech transfer and manufacture GS-100, a recombinant AAV9 vector and a suspension process AAV NGLY1 gene therapy for Phase I/II/III clinical trial material for NGLY1 Deficiency.

                            Brand Name : GS-100

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            March 25, 2024

                            Lead Product(s) : GS-100

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Recipient : Andelyn Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            Lead Product(s) : GS-100

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Grace Science Announces First Patient Treated in Phase 1/2/3 Trial of AAV9 Gene Therapy

                            Details : GS-100 is a recombinant, single-stranded AAV9 vector that encodes a full-length version of the human NGLY1 gene. It is being evaluated for the treatment of NGLY1 Deficiency.

                            Brand Name : GS-100

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            March 05, 2024

                            Lead Product(s) : GS-100

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Lead Product(s) : AAV9 Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Study Phase : Undisclosed

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Grace Science Receives U.S. FDA Orphan Drug Designation for GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency

                            Details : Grace Science is advancing GS-100 into a first-in-human clinical trial for NGLY1 deficiency. The viral vector development and clinical manufacturing services for GS-100 will be provided by Thermo Fisher Scientific at its site in Alachua, FL.

                            Brand Name : GS-100

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            June 22, 2021

                            Lead Product(s) : AAV9 Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Undisclosed

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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