[{"orgOrder":0,"company":"Mustang Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mustang Bio Receives Advanced Therapy Medicinal Product Classification from EMA for MB-107 Lentiviral Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mustang Bio Announces Rare Pediatric Disease Designation for MB-107 for the Treatment of X-linked Severe Combined Immunodeficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mustang Bio Announces Orphan Drug Designation for MB-107 to Treat X-linked Severe Combined Immunodeficiency in Newly Diagnosed Infants","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Minaris Regenerative Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Mustang Bio and Minaris Regenerative Medicine Sign Technology Transfer and GMP Manufacturing Deal for MB-107 Lentiviral Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mustang Bio Provides Updates on its Lentiviral Gene Therapies for the Treatment of X-linked Severe Combined Immunodeficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Presents Data Supporting the Initiation of the EDIT-301 Phase 1\/2 RUBY Clinical Trial for the Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Reports on Recent Progress and Outlook at J.P. 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Find Clinical Drug Pipeline Developments & Deals for Busulfan
PTCy (post-transplant cyclophosphamide) in combination with Busulfan, Fludarabine & Tacrolimus is being evaluated for the treatment of patients with blood cancers who have received peripheral blood stem cells from partially matched unrelated donors.
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.
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.
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).
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).
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.
MB-207 has been studied at NIAID since 2012 and continues to be assessed in a NIAID-supported Phase 1/2 clinical trial for XSCID in patients over the age of two who have received prior HSCT.
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.
EDIT-301 is the first experimental medicine generated using CRISPR/Cas12a gene editing. The Company reported the data in an oral presentation at the 26th Congress of the European Hematology Association (EHA) being held virtually.
FDA removes clinical hold for pivotal Phase 2 MB-107 clinical trial. Mustang will proceed with its plans to initiate the pivotal Phase 2 trial in newly diagnosed XSCID patients.