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    [{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Completion of IND-enabling Safety Studies for FTX-6058 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Preclinical Proof-of-Concept Data for FTX-6058 at the Virtual Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Multiple Presentations During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Presents Published Structure of Investigational Small Molecule FTX-6058 at the ACS Spring 2021 Virtual Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics to Present Initial Data from Phase 1b Trial of FTX-6058 in Adults Living with Sickle Cell Disease at the European Hematology Association (EHA) Hybrid Congress in Vienna, Austria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Proof-of-Concept for FTX-6058 in Sickle Cell Disease Based on Initial Data from the Ongoing Phase 1b Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Clinical Hold on FTX-6058 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"}]

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics Announces Clinical Hold on FTX-6058 in Sickle Cell Disease

              Details:

              FTX-6058 is an investigational oral small-molecule inhibitor of embryonic ectoderm development which leads to potent downregulation of key fetal globin repressors, including BCL11A, thereby causing an increase in fetal hemoglobin.

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: FTX-6058

              Highest Development Status: Phase I Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 24, 2023

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics Announces Proof-of-Concept for FTX-6058 in Sickle Cell Disease Based on Initial Data from the Ongoing Phase 1b Trial

              Details:

              Data from first subjects to receive a 6 mg dose of FTX-6058, an oral HbF inducer, showed a rapid and robust induction of HbF, and subjects achieved increases of up to 6.3% over baseline.

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: FTX-6058

              Highest Development Status: Phase I Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable June 10, 2022

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics to Present Initial Data from Phase 1b Trial of FTX-6058 in Adults Living with Sickle Cell Disease at the European Hematology Association (EHA) Hybrid C...

              Details:

              FTX-6058 is an investigational oral small-molecule Embryonic Ectoderm Development Inhibitor (EEDI) that has demonstrated robust induction of fetal hemoglobin (HbF) in human cells and animal models of sickle cell disease (SCD).

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: FTX-6058

              Highest Development Status: Phase I Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable May 12, 2022

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics Presents Published Structure of Investigational Small Molecule FTX-6058 at the ACS Spring 2021 Virtual Conference

              Details:

              FTX-6058 is a highly potent orally bioavailable small molecule EED inhibitor for the potential treatment of select hemoglobinopathies, including sickle cell disease and β-thalassemia.

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: FTX-6058

              Highest Development Status: Phase I Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable April 09, 2021

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics Announces Multiple Presentations During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition

              Details:

              Multiple posters on the company’s IND-supporting preclinical studies with its Embryonic Ectoderm Development (EED) inhibitor, FTX-6058, for hemoglobinopathies such as sickle cell disease and β-thalassemia will be presented at the virtual 62nd ASH Annual Meeting and Exposition.

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: FTX-6058

              Highest Development Status: Phase I Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable November 04, 2020

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease

              Details:

              This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts. Part A will be a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study in up to six cohorts.

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: FTX-6058

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable October 05, 2020

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics Announces Preclinical Proof-of-Concept Data for FTX-6058 at the Virtual Annual Sickle Cell Disease Research & Educational Symposium and 43rd National S...

              Details:

              Fulcrum has identified FTX-6058, a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models.

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: FTX-6058

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable September 25, 2020

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              Fulcrum Therapeutics

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              Fulcrum Therapeutics Announces Completion of IND-enabling Safety Studies for FTX-6058 in Sickle Cell Disease

              Details:

              Completed preclinical studies illustrate that FTX-6058, has the potential to provide distinct advantages over biologics and gene therapies being used for the treatment of sickle cell disease.

              Lead Product(s): FTX-6058

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

              Highest Development Status: IND Enabling Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable April 13, 2020

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              Boai NKY Pharmaceuticals Ltd. is one of the largest global PVP suppliers.
              Renejix: CDMO with expertise in small molecule oral dosage, delivery tech, and multi-modality manufacturing.
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