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[{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA offers CRISPR, Vertex drug program VIP status for its comeback play","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"CRISPR Therapeutics"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Fast Track Designation for BCX9930 in PNH","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small 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molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Forms Scientific Advisory Board to Guide Global Development of CAN106 in Complement-mediated Diseases","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Deep Track Capital","pharmaFlowCategory":"D","amount":"$135.0 million","upfrontCash":"Undisclosed","newsHeadline":"Hemab Therapeutics Announces $135M Series B Financing to Advance First Prophylactic Treatments for Underserved Bleeding and Thrombotic Disorders","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"DENMARK","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Hemab Therapeutics"},{"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":"Cell and Gene therapy","graph2":"Editas Medicine"},{"orgOrder":0,"company":"Azzur Group","sponsor":"Editas Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Editas Medicine and Azzur Group Expand Partnership to Accelerate Editas\u2122 Manufacturing Capabilities for Advancing the EDIT-301 Program through Approval to Commercialization","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Azzur Group"},{"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":"Cell and Gene therapy","graph2":"Editas Medicine"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Kamau Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Kamau Therapeutics Emerges from Stealth With Clinical-Stage Novel Gene Correction Technology Targeting Life-Threatening Genetic Diseases","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Graphite Bio"}]

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            Details:

            Under the agreement, Kamau will focus on the clinical development of lead program Nula-Cel (nulabeglogene autogedtemcel), which is being evaluated in Phase I/II clinical trial studies for the treatment of sickle cell disease.

            Lead Product(s): Nulabeglogene Autogedtemcel

            Therapeutic Area: Hematology Product Name: Nula-Cel

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

            Partner/Sponsor/Collaborator: Kamau Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement December 07, 2023

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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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            The partnersip aims to support the scaling of EDIT-301, Editas Medicine’s experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), from approval to commercialization.

            Lead Product(s): EDIT-301

            Therapeutic Area: Hematology Product Name: EDIT-301

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

            Partner/Sponsor/Collaborator: Editas Medicine

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership July 27, 2023

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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 April 27, 2023

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            The Funding enables completion of ongoing Phase 1/2 study of bispecific antibody HMB-001 in Glanzmann Thrombasthenia, initiation of pivotal studies, start and conclusion of Phase 1/2 study of HMB-VWF in von Willebrand Disease, as well as future pipeline evolution.

            Lead Product(s): HMB-001

            Therapeutic Area: Hematology Product Name: HMB-001

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Deep Track Capital

            Deal Size: $135.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing February 20, 2023

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            CAN106 achieved rapid, dose-dependent reductions in both free C5 complement, a measure of serum hemolytic activity, within 24 hours of dosing. All subjects in two highest dose groups of CAN106 showed >99% reduction in free C5 and ≥90% inhibition of CH50.

            Lead Product(s): CAN106

            Therapeutic Area: Hematology Product Name: CAN106

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 06, 2022

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            The FDA has granted both Fast Track status and Orphan Drug Designation to BCX9930 for PNH. BioCryst has confirmed meetings with regulators in the 4th quarter of 2020 to discuss the advanced development program for BCX9930.

            Lead Product(s): BCX9930

            Therapeutic Area: Hematology Product Name: BCX9930

            Highest Development Status: Phase I/ Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 30, 2020

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            BioCryst Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

            Lead Product(s): BCX9930

            Therapeutic Area: Hematology Product Name: BCX9930

            Highest Development Status: Phase I/ Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 31, 2020

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            U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

            Lead Product(s): BCX9930

            Therapeutic Area: Hematology Product Name: BCX9930

            Highest Development Status: Phase I/ Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 03, 2020

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            CTX001 has received Orphan Drug Designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the EMA for sickle cell disease and transfusion-dependent beta thalassemia .

            Lead Product(s): CTX001

            Therapeutic Area: Hematology Product Name: Undisclosed

            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 11, 2020

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