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    [{"orgOrder":0,"company":"NMDP","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Cytotoxic Drug","year":"2024","type":"Inapplicable","leadProduct":"Cyclophosphamide","moa":"||DNA","graph1":"Oncology","graph2":"Phase II","graph3":"NMDP","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"NMDP \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"NMDP \/ Inapplicable"},{"orgOrder":0,"company":"Center for International Blood and Marrow Transplant Research","sponsor":"National Marrow Donor Program | Incyte Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Busulfan","moa":"DNA","graph1":"Oncology","graph2":"Phase II","graph3":"Center for International Blood and Marrow Transplant Research","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Center for International Blood and Marrow Transplant Research \/ National Marrow Donor Program | Incyte Corporation","highestDevelopmentStatusID":"8","companyTruncated":"Center for International Blood and Marrow Transplant Research \/ National Marrow Donor Program | Incyte Corporation"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Busulfan","moa":"||CD34+","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mustang Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Inapplicable"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mustang Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Inapplicable"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mustang Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Inapplicable"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mustang Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Inapplicable"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mustang Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Inapplicable"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Busulfan","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mustang Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Inapplicable"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Minaris Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"MB-107","moa":"||IL-2-gamma receptor","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Mustang Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Mustang Bio \/ Minaris Regenerative Medicine","highestDevelopmentStatusID":"7","companyTruncated":"Mustang Bio \/ Minaris Regenerative Medicine"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"BEAM-101","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Beam Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Beam Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Essen Biotech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Busulfan","moa":"DNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Essen Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Essen Biotech \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Essen Biotech \/ Inapplicable"}]

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                            01

                            Center for International Blood and Marrow Transplant Research

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                            Center for International Blood and Marrow Transplant Research

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                            Lead Product(s) : Busulfan

                            Therapeutic Area : Oncology

                            Study Phase : Phase II

                            Sponsor : National Marrow Donor Program | Incyte Corporation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxi...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 05, 2025

                            Lead Product(s) : Busulfan

                            Therapeutic Area : Oncology

                            Highest Development Status : Phase II

                            Sponsor : National Marrow Donor Program | Incyte Corporation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Beam Therapeutics

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                            Lead Product(s) : BEAM-101,Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Patient Death Taints Otherwise Positive Data for Beam’s Sickle Cell Gene Therapy

                            Details : BEAM-101 is a cell therapy which increases the production of fetal hemoglobin (HbF), was being investigated in patients with sickle cell disease.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 11, 2024

                            Lead Product(s) : BEAM-101,Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Essen Biotech

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                            Lead Product(s) : Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 03, 2024

                            Lead Product(s) : Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            NMDP

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                            Lead Product(s) : Cyclophosphamide,Busulfan,Fludarabine Phosphate

                            Therapeutic Area : Oncology

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NMDP Announces Enrollment of First Patient in OPTIMIZE Clinical Trial

                            Details : PTCy (post-transplant cyclophosphamide) combined with Busulfan, Fludarabine, and Tacrolimus is being evaluated for blood cancer patients receiving stem cells from partially matched unrelated donors.

                            Product Name : PTCy

                            Product Type : Cytotoxic Drug

                            Upfront Cash : Inapplicable

                            February 20, 2024

                            Lead Product(s) : Cyclophosphamide,Busulfan,Fludarabine Phosphate

                            Therapeutic Area : Oncology

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Editas Medicine

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                            Editas Medicine

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                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EDIT-301 for the Tre...

                            Details : EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, which demonstrated a sustained increase in fetal hemoglobin production in patients with with severe SCD and TDT.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            October 16, 2023

                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Editas Medicine

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                            Editas Medicine

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                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Editas Medicine Receives FDA Orphan Drug Designation for EDIT-301 for the Treatment of Sickle Cell Disease

                            Details : EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, which demonstrated a sustained increase in fetal hemoglobin production in patients with with severe SCD and TDT...

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 27, 2023

                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Editas Medicine

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                            Editas Medicine

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                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Editas Medicine Reports on Recent Progress and Outlook at J.P. Morgan Healthcare Conference

                            Details : EDIT-301 is an experimental cell therapy medicine currently being investigated in a RUBY trial, a clinical study in patients with severe SCD and TDT. After clearance from IND company will initiate the Phase 1/2 study for EDIT-301 in patients with TDT.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            October 01, 2022

                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Editas Medicine

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                            Editas Medicine

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                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Editas Medicine Announces Clinical Achievements in the Development of EDIT-301 for Sickle Cell Disease

                            Details : EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            July 27, 2022

                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Editas Medicine

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                            Editas Medicine

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                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Editas Medicine Announces Positive Safety and Efficacy Data from the First Two Patients Treated in the RUBY Tr...

                            Details : EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            June 12, 2022

                            Lead Product(s) : Renizgamglogene Autogedtemcel,Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Mustang Bio

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                            Mustang Bio

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                            Lead Product(s) : MB-107,Busulfan

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Mustang Bio Announces Data on Treatment with Lentiviral Viral Vector Gene Therapy for X-Linked Severe Combined...

                            Details : All 23 treated patients are alive and 20 patients with follow-up greater than 4 months recovered from pre-existing infections, are off protective isolation and prophylactic antimicrobials, and have normal growth velocity.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 05, 2022

                            Lead Product(s) : MB-107,Busulfan

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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