[{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"OXB","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Cell and 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":"Oral 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 and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"BAY2599023","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":"Intravenous Infusion","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 and 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":"Intravenous Infusion","sponsorNew":"Sirion Biotech \/ Mustang Bio","highestDevelopmentStatusID":"7","companyTruncated":"Sirion Biotech \/ Mustang Bio"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          Top Deals by Deal Size (USD bn)

                          01

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          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 : BI 3720931

                          Product Type : Cell and 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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                          02

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          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 : DTX201

                          Product Type : Cell and 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

                          Deal Type : Inapplicable

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                          03

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          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 : MB-207

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          June 10, 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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