[{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CRISPR Therapeutics Provides Research Update for CTX120","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"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":"Hematology","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CRISPR Therapeutics Provides Business Update and Reports Second Quarter 2020 Financial Results","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CRISPR Therapeutics and Vertex Pharmaceuticals' CTX001\u2122 Gets PRIME Designation Granted by the EMA 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":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex to Present CRISPR\/Cas9 Gene-Editing Therapy CTX001\u2122 Data for Severe Hemoglobinopathies at the 62nd ASH Meeting","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":"CRISPR Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","amount":"$1,100.0 million","upfrontCash":"$900.0 million","newsHeadline":"Vertex Pharmaceuticals and CRISPR Therapeutics Amend Collaboration for CTX001\u2122 in Sickle Cell Disease and Beta Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex and CRISPR Therapeutics Announce PRIME Designation Granted by the EMA to CTX001\u2122 for Transfusion-Dependent Beta Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex and CRISPR Therapeutics to Present New Clinical Data on Investigational CTX001 For Severe Hemoglobinopathies at EHA2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Complement Therapeutics","sponsor":"Gimv","pharmaFlowCategory":"D","amount":"$78.9 million","upfrontCash":"Undisclosed","newsHeadline":"Complement Therapeutics Secures \u20ac72 Million in Series A Financing to Advance Novel Therapies Targeting Complement-Mediated Diseases","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Ophthalmology","graph2":"Preclinical"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"CRISPR Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex and CRISPR Therapeutics Announce Acceptance of Late-Breaking Abstract for CTX001\u2122 at the 2022 Annual European Hematology Association (EHA) Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]
Find Clinical Drug Pipeline Developments & Deals for CTX001
With the financing, CTx will continue the development and complete a Phase Ib clinical proof of concept of its lead product CTx001. CTx001 is a highly innovative AAV gene therapy for the treatment of Geograpic Atrophy (GA) secondary to dry age-related macular degeneration.
Exa‑cel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 gene‑edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent VOCs.
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD.
Vertex will lead global development, manufacturing and commercialization of CTX001 with support from CRISPR Therapeutics. Vertex will be responsible for 60% of program costs and will receive 60% of profits from future sales of CTX001 worldwide.
Data in seven patients from two ongoing Phase 1/2 trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition.
European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).
CRISPR Therapeutics continues to make substantial progress driving its multiple, ongoing clinical development programs. Enrollment in its immuno-oncology trials is ongoing, the compay has re-initiated dosing in its CTX001 trials.
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 .
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