Pipeline

Hemoglobinopathies

Hemoglobinopathies

 
Research
Ind-Enabling
Clinical
Approved
 
  • * Exagamglogene Autotemcel (exa-cel): Sickle cell disease (SCD)
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    Description: Autologous, ex vivo CRISPR/Cas9 gene-edited therapy in which aims to edit a patient’s own hematopoietic stem cells to produce fetal hemoglobin in red blood cells. Exa-cel, a CRISPR/Cas9 gene-edited therapy arising out of our collaboration with Vertex Pharmaceuticals Incorporated, has now been approved in some countries for certain eligible patients with sickle cell disease or transfusion-dependent beta thalassemia. Vertex is the manufacturer and exclusive license holder of exa-cel.

    Structure: Collaboration with Vertex

  • * Exagamglogene Autotemcel (exa-cel): β-thalassemia
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    Description: Autologous, ex vivo CRISPR/Cas9 gene-edited therapy in which aims to edit a patient’s own hematopoietic stem cells to produce fetal hemoglobin in red blood cells. Exa-cel, a CRISPR/Cas9 gene-edited therapy arising out of our collaboration with Vertex Pharmaceuticals Incorporated, has now been approved in some countries for certain eligible patients with sickle cell disease or transfusion-dependent beta thalassemia. Vertex is the manufacturer and exclusive license holder of exa-cel.

    Structure: Collaboration with Vertex

  • Next-generation conditioning
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    Description: Targeted conditioning agent to improve the safety of hematopoietic stem cell transplant

    Structure: Wholly owned (collaboration with Vertex for applications in β-thalassemia and SCD)

  • In vivo editing of HSCs
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    Description: Enabling delivery of CRISPR/Cas9 to hematopoietic stem cells (HSCs) in vivo to bypass the need for ex vivo gene editing and hematopoietic stem cell transplant

    Structure: Wholly owned (collaboration with Vertex for applications in β-thalassemia and SCD)

Screen Shot 2023 11 16 at 8 15 57 AM

*Currently approved in some countries for certain eligible people.

Immuno oncology

Immuno-Oncology & Autoimmune Disease

 
Research
Ind-Enabling
Clinical
Approved
 
  • CTX112: Anti-CD19 allogeneic CAR T
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    Description: Investigational allogeneic CRISPR/Cas9 gene-edited CAR T cell therapy in development for the treatment of CD19+ malignancies and autoimmune diseases that incorporates novel edits designed to enhance CAR T potency and reduce CAR T exhaustion

    Structure: Wholly owned

  • CTX131: Anti-CD70 allogeneic CAR T
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    Description: Investigational allogeneic CRISPR/Cas9 gene-edited CAR T cell therapy in development for the treatment of solid tumors and hematological malignancies that incorporates novel edits designed to enhance CAR T potency and reduce CAR T exhaustion

    Structure: Wholly owned

  • Anti-GPC3 autologous CAR-T
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    Description: Autologous CRISPR/Cas9 gene-edited CAR T cell therapy in development for hepatocellular carcinoma

    Structure: Collaboration with Roswell Park Comprehensive Cancer Center (CRISPR retains commercial rights)

  • Anti-CD70 allogeneic CAR-NK
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    Description: Investigational allogeneic CRISPR/Cas9 gene-edited CAR-NK cell therapy

    Structure: Collaboration with Nkarta

  • Other CAR-T Programs
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    Description: Investigational CRISPR/Cas9 gene-edited CAR T cell therapies that incorporate novel edits designed to enhance CAR T potency and reduce CAR T exhaustion

    Structure: Wholly owned

In Vivo

In Vivo Approaches

 
Research
Ind-Enabling
Clinical
Approved
 
Regenative Medicine

Regenerative Medicine

 
Research
Ind-Enabling
Clinical
Approved
 
DNA

Other Disclosed Partnered Programs

 
Research
Ind-Enabling
Clinical
Approved
 

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CTX112™, CTX131™, CTX310™, CTX320™, CTX330™, and CTX211™ are trademarks and registered trademarks of CRISPR Therapeutics AG.

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