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    [{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"OXB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BI 3720931","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Boehringer Ingelheim GmbH \/ OXB","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ OXB"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"BAY2599023","moa":"Coagulation factor VIII (F8)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Bayer AG \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"BI3720931","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"BI3720931","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Boehringer Ingelheim GmbH \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"DTX201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Bayer AG \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Sirion Biotech","sponsor":"Mustang Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell & Gene Therapy","year":"2020","type":"Licensing Agreement","leadProduct":"MB-207","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sirion Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sirion Biotech \/ Mustang Bio","highestDevelopmentStatusID":"7","companyTruncated":"Sirion Biotech \/ Mustang Bio"}]

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                            01

                            Boehringer Ingelheim GmbH

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                            Lead Product(s) : BI3720931

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Stud...

                            Details : BI3720931 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            May 08, 2025

                            Lead Product(s) : BI3720931

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Boehringer Ingelheim GmbH

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                            Lead Product(s) : BI 3720931

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : OXB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            OXB’s Technology to Support Boehringer’s Gene Therapy for Cystic Fibrosis

                            Details : BI 3720931 is a first-in-class, inhaled lentiviral vector-based gene therapy designed to insert a functional copy of the CFTR gene in the DNA of airway epithelial cells for treating cystic fibrosis.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            February 20, 2025

                            Lead Product(s) : BI 3720931

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : OXB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Boehringer Ingelheim GmbH

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                            Lead Product(s) : BI3720931

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic F...

                            Details : BI3720931 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            July 23, 2024

                            Lead Product(s) : BI3720931

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Sirion Biotech

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                            Lead Product(s) : MB-207

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Mustang Bio

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            Mustang Bio Licenses LentiBOOST™ Technology from SIRION Biotech

                            Details : Mustang has acquired rights to SIRION’s LentiBOOST™ technology for the development of MB-207, Mustang’s lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            October 06, 2020

                            Lead Product(s) : MB-207

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Mustang Bio

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            05

                            Bayer AG

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                            Lead Product(s) : BAY2599023

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Bayer's Gene Therapy BAY 2599023 Showing Steady FVIII Activity, Safety in Trial

                            Details : Two of the six patients treated to date in this clinical study, both given the therapy at lowest dose, are still showing FVIII activity at more than one year post-treatment, its investigators report.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            July 24, 2020

                            Lead Product(s) : BAY2599023

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

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                            Bayer AG

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                            Lead Product(s) : DTX201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 ...

                            Details : DTX201 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            July 17, 2018

                            Lead Product(s) : DTX201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Ultragenyx Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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