Seqens Seqens

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[{"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. Morgan Healthcare Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","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 an Update on its IND Application for MB-207, a Lentiviral Gene Therapy for Treatment of X-linked Severe Combined Immunodeficiency (\u201cXSCID\u201d) in Previously Transplanted Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","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 Data on Treatment with Lentiviral Viral Vector Gene Therapy for X-Linked Severe Combined Immunodeficiency Selected for Oral Presentation at American Society of Gene & Cell Therapy 25th Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","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 Announces Clinical Achievements in the Development of EDIT-301 for 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":"July 2022","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 Announces Positive Safety and Efficacy Data from the First Two Patients Treated in the RUBY Trial of EDIT-301 for the Treatment of Severe 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":"December 2022","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 Receives FDA Orphan Drug Designation for EDIT-301 for the Treatment of Sickle Cell Disease","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Hematology","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EDIT-301 for the Treatment of Severe Sickle Cell Disease","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Hematology","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"NMDP","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NMDP Announces Enrollment of First Patient in OPTIMIZE Clinical Trial","therapeuticArea":"Oncology","highestDevelopmentStatus":"Phase II","country":"","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Oncology","graph2":"Phase II"}]

Find Clinical Drug Pipeline Developments & Deals for MolPort-001-783-406

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            Details:

            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.

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

            Therapeutic Area: Oncology Product Name: PTCy

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 20, 2024

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            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.

            Lead Product(s): EDIT-301,Busulfan

            Therapeutic Area: Hematology Product Name: EDIT-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 16, 2023

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            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.

            Lead Product(s): EDIT-301,Busulfan

            Therapeutic Area: Hematology Product Name: EDIT-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 27, 2023

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            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).

            Lead Product(s): EDIT-301,Busulfan

            Therapeutic Area: Genetic Disease Product Name: EDIT-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 06, 2022

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            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).

            Lead Product(s): EDIT-301,Busulfan

            Therapeutic Area: Genetic Disease Product Name: EDIT-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 27, 2022

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            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.

            Lead Product(s): MB-107,Busulfan

            Therapeutic Area: Genetic Disease Product Name: MB-107

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 03, 2022

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            Details:

            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.

            Lead Product(s): MB-207,Palifermin,Busulfan

            Therapeutic Area: Genetic Disease Product Name: MB-207

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 24, 2022

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            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.

            Lead Product(s): EDIT-301,Busulfan

            Therapeutic Area: Genetic Disease Product Name: EDIT-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 10, 2022

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            Details:

            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.

            Lead Product(s): EDIT-301,Busulfan

            Therapeutic Area: Genetic Disease Product Name: EDIT-301

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 11, 2021

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            Details:

            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.

            Lead Product(s): MB-107,Busulfan

            Therapeutic Area: Genetic Disease Product Name: MB-107

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 02, 2021

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