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II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MeiraGTx \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"7","companyTruncated":"MeiraGTx \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"AAV-CNGB3","moa":"Cyclic nucleotide-gated channel beta-3 (CNGB3)","graph1":"Ophthalmology","graph2":"Phase I\/ Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MeiraGTx \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"7","companyTruncated":"MeiraGTx \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Syne Qua Non Limited | EMAS Pharma","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"AAV-CNGB3","moa":"Cyclic nucleotide-gated channel beta-3 (CNGB3)","graph1":"Ophthalmology","graph2":"Phase I\/ Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MeiraGTx \/ Syne Qua Non Limited | EMAS Pharma","highestDevelopmentStatusID":"7","companyTruncated":"MeiraGTx \/ Syne Qua Non Limited | EMAS Pharma"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Aav-Gad","moa":"Glutamic acid decarboxylase 1 (GAD1)","graph1":"Neurology","graph2":"Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Aav-Gad","moa":"Glutamic acid decarboxylase 1 (GAD1)","graph1":"Neurology","graph2":"Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Hologen","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Collaboration","leadProduct":"AAV-GAD","moa":"Glutamic acid decarboxylase 1 (GAD1)","graph1":"Neurology","graph2":"Phase II","graph3":"MeiraGTx","amount2":0.42999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0.42999999999999999,"dosageForm":"Infusion","sponsorNew":"MeiraGTx \/ Hologen","highestDevelopmentStatusID":"8","companyTruncated":"MeiraGTx \/ Hologen"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"AAV-GAD","moa":"Glutamic acid decarboxylase 1 (GAD1)","graph1":"Neurology","graph2":"Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene 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Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ MeiraGTx","highestDevelopmentStatusID":"7","companyTruncated":"MeiraGTx \/ MeiraGTx"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Botaretigene Sparoparvovec","moa":"GTPase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2016","type":"Inapplicable","leadProduct":"AAV-RPE65","moa":"Retinal pigment epithelium protein (RPE65)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Otolaryngology (Ear, Nose, Throat)","country":"U.S.A","productType":"Undisclosed","year":"2019","type":"Inapplicable","leadProduct":"Aav2Haqp1","moa":"Undisclosed","graph1":"Otolaryngology (Ear, Nose, Throat)","graph2":"Phase I","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Otolaryngology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"National Institute of Dental and Craniofacial Research","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"AAV2-hAQP1","moa":"Undisclosed","graph1":"Oncology","graph2":"Phase I","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Infusion","sponsorNew":"MeiraGTx \/ National Institute of Dental and Craniofacial Research","highestDevelopmentStatusID":"6","companyTruncated":"MeiraGTx \/ National Institute of Dental and Craniofacial Research"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Dental and Oral Health","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"AAV2-hAQP1","moa":"Undisclosed","graph1":"Dental and Oral Health","graph2":"Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Dental & Oral Health","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Dental and Oral Health","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"AAV2-hAQP1","moa":"Undisclosed","graph1":"Dental and Oral Health","graph2":"Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Dental & Oral Health","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MeiraGTx \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"MeiraGTx \/ Undisclosed"},{"orgOrder":0,"company":"MeiraGTx","sponsor":"Syne Qua Non Limited | Bionical Emas","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2017","type":"Inapplicable","leadProduct":"AAV5-RPGR","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"MeiraGTx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"MeiraGTx \/ Syne Qua Non Limited | Bionical Emas","highestDevelopmentStatusID":"7","companyTruncated":"MeiraGTx \/ Syne Qua Non Limited | Bionical Emas"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : The collaboration aims to advance the clinical development of AAV-GAD, which is being evaluated in the mid-stage clinical trial studies for the treatment of Parkinson’s disease.

                          Product Name : AAV-GAD

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : $200.0 million

                          March 17, 2025

                          Lead Product(s) : AAV-GAD

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase II

                          Sponsor : Hologen

                          Deal Size : $430.0 million

                          Deal Type : Collaboration

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                          02

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : AAV-GAD is a gene therapy for Parkinson's disease. It delivers the glutamic acid decarboxylase (GAD) gene to the subthalamic nucleus, to increase production of the inhibitory neurotransmitter GABA.

                          Product Name : AAV-GAD

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          October 15, 2024

                          Lead Product(s) : AAV-GAD

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : AAV2-hAQP1 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Xerostomia.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          August 09, 2024

                          Lead Product(s) : AAV2-hAQP1

                          Therapeutic Area : Dental and Oral Health

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Under the agreement, Janssen gains the remaining interests in investigational gene therapy AAV5-RPGR (botaretigene sparoparvovec) for the treatment of X-linked retinitis pigmentosa.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : $350.0 million

                          December 21, 2023

                          Lead Product(s) : Botaretigene Sparoparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Johnson & Johnson Innovative Medicine

                          Deal Size : $415.0 million

                          Deal Type : Agreement

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                          05

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Sanofi will receive a right of first negotiation for the use of MeiraGTx’s Riboswitch gene regulation technology for certain Immunology and Inflammation and CNS targets, including AAV-AQP1, a gene therapy for xerostomia caused by Sjogren's syndrome.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Undisclosed

                          October 30, 2023

                          Lead Product(s) : AAV-AQP1

                          Therapeutic Area : Dental and Oral Health

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Sanofi

                          Deal Size : $30.0 million

                          Deal Type : Financing

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                          06

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : AAV2-hAQP1 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Xerostomia.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          July 03, 2023

                          Lead Product(s) : AAV2-hAQP1

                          Therapeutic Area : Dental and Oral Health

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Aav-Gad is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Parkinson Disease.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          June 08, 2023

                          Lead Product(s) : Aav-Gad

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Aav-Gad is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Parkinson Disease.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          November 02, 2022

                          Lead Product(s) : Aav-Gad

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile and efficacy assessments in this proof-of-concept study demonstrated improvements in retinal sensitivity, visual function and functional vision.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          October 01, 2022

                          Lead Product(s) : Botaretigene Sparoparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : One-year findings support advancement into Phase 3 and include significant functional vision improvements observed in the time taken to walk through a vision-guided mobility maze at nine months.

                          Product Name : AAV-RPGR

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          November 13, 2020

                          Lead Product(s) : Botaretigene Sparoparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Johnson & Johnson Innovative Medicine

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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