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United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia","therapeuticArea":"Hematology","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Hematology","graph2":"Approved"}]
Find Clinical Drug Pipeline Developments & Deals by CRISPR Therapeutics
The net proceeds will be used for the development of CTX112, which is an investigational allogeneic CRISPR/Cas9 gene-edited CAR T cell therapy for the treatment of CD19+ malignancies and autoimmune diseases.
Casgevy (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
Exa-cel, formerly known as CTX001™, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with SCD or TDT.
Exa-cel (exagamglogene autotemcel), formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with sickle cell disease.
Exa-cel, formerly known as CTX001™, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with SCD or TDT.
Exa-cel, formerly known as CTX001™, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with SCD or TDT.
Exa-cel, formerly known as CTX001™, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with SCD or TDT.
Under the agreement, Vertex will receive non-exclusive rights to CRISPR Therapeutics’ CRISPR/Cas9 to accelerate development of potentially curative cell therapies for T1D and generating fully differentiated, insulin-producing hypoimmune islet cells for T1D.
Lead Product(s):
Insulin-producing Hypoimmune Islet Cells
CTX112 consists anti-CD19 allogeneic CRISPR-Cas9-engineered T cells, it is being investigated in subjects with relapsed or refractory B-cell malignancies.
Exa-cel (Exagamglogene Autotemcel), 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.
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