[{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"rAAV2tYF-PR1.7-hCNGB3","moa":"Undisclosed","graph1":"Ophthalmology","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"CNGB3","moa":"CNGB3","graph1":"Ophthalmology","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Ophthalmology","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Neurology","graph2":"Preclinical","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intra-cisterna magna Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Ophthalmology","graph2":"IND Enabling","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AGTC-501","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"SparingVision","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ SparingVision","highestDevelopmentStatusID":"5","companyTruncated":"Agtc \/ SparingVision"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Cantor Fitzgerald","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0.01,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Cantor Fitzgerald","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Cantor Fitzgerald"},{"orgOrder":0,"company":"Agtc","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ H.C. Wainwright & Co","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ H.C. Wainwright & Co"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0.02,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Syncona Limited"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-RPGR","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"TeamedOn","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"rAAV2tYF-CB-hRS1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ TeamedOn","highestDevelopmentStatusID":"1","companyTruncated":"Agtc \/ TeamedOn"}]

Find Clinical Drug Pipeline Developments & Deals by Agtc

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                          Top Deals by Deal Size (USD bn)

                          01

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : SKYLINE Trial of AGTC-501 (rAAV2tYF-GRK1-hRPGRco), on track to report three-month interim study results, including visual acuity, visual sensitivity, mobility and safety data, in Q2 2022 for the treatment of X-linked retinitis pigmentosa.

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 01, 2022

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : On closing, AGTC will be Syncona’s third company focused on retinal gene therapy, and transitioning AGTC-501 to Syncona’s experienced stewardship with the goal of advancing this differentiated product candidate to patients with XLRP.

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : $23.5 million

                          October 23, 2022

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Syncona Limited

                          Deal Size : $23.5 million

                          Deal Type : Acquisition

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                          03

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : AGTC intends to use the net proceeds from this offering, to fund its ongoing Skyline and Vista clinical trials in its X-linked retinitis pigmentosa (XLRP) program, AGTC-501 (rAAV2tYF-GRK1-hRPGRco) and its ongoing Phase 1/2 clinical trials in its Achromat...

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 13, 2022

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : H.C. Wainwright & Co

                          Deal Size : Undisclosed

                          Deal Type : Public Offering

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                          04

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : AGTC-501, recombinant AAV viral vector-based gene therapy targeting mutations in RPGR gene in patients with X-linked retinitis pigmentosa, demonstrated 62.5% of patients in dose group B were responders for improvements in visual sensitivity, study’s pr...

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 16, 2022

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : 18 months after treatment, of eyes in study with visible foveal EZ at baseline underwent subretinal administration of AGTC-501 (rAAV2tYF-GRK1-hRPGRco), showed recovery of foveal EZ and nearly half had improved EZ appearance, correlated with improvement i...

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 05, 2022

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : AGTC-401, a recombinant AAV viral vector-based gene therapy targeting mutations in the CNGB3 gene in patients with achromatopsia demonstrated a favorable safety profile through second-highest dose (1.1E+12 vg/mL) in ACHMB3 pediatric patients.

                          Product Name : AGTC-401

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 04, 2022

                          Lead Product(s) : rAAV2tYF-PR1.7-hCNGB3

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : AGTC intends to use the net proceeds from this offering, together with other available funds, to fund its ongoing Skyline and Vista clinical trials in its X-linked retinitis pigmentosa (XLRP) program and its ongoing Phase 1/2 clinical trials in its Achro...

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          March 22, 2022

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Cantor Fitzgerald

                          Deal Size : $9.8 million

                          Deal Type : Public Offering

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                          08

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : Administration of AVrh10-Based Gene Therapy, in non-human primates resulted in a dose-dependent and sustained expression of human progranulin in cerebrospinal fluid and achieved levels above physiological level in normal humans, without any vector-associ...

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          February 05, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Neurology

                          Highest Development Status : Preclinical

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : Adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), particularly dry-age related macular degeneration (AMD), including the relevance of expressing complement factor H as a therapeutic approach.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          October 19, 2021

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : IND Enabling

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Vietnam Medi-Pharm Expo
                          Not Confirmed
                          Vietnam Medi-Pharm Expo
                          Not Confirmed

                          Details : Adeno-associated virus (AAV)-based gene therapies, subretinal gene therapy used for treatment of Achromatopsia, and AGTC-501 for X-Linked Retinitis Pigmentosa.

                          Product Name : AGTC-501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 28, 2021

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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