Pipeline
Hemoglobinopathies
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* Exagamglogene Autotemcel (exa-cel): Sickle cell disease (SCD)
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
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* Exagamglogene Autotemcel (exa-cel): β-thalassemia
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
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Next-generation conditioning
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)
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In vivo editing of HSCs
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)
*Currently approved in some countries for certain eligible people.
Immuno-Oncology
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CTX112: Anti-CD19 allogeneic CAR T
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
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CTX131: Anti-CD70 allogeneic CAR T
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
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Anti-CD70 allogeneic CAR-NK
Description: Investigational allogeneic CRISPR/Cas9 gene-edited CAR-NK cell therapy
Structure: Collaboration with Nkarta
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CTX121: Anti-BCMA allogeneic CAR-T
Description: Allogeneic CRISPR/Cas9 gene-edited CAR T cell therapy in development for multiple myeloma that incorporates additional edits designed to enhance CAR T potency and reduce CAR T exhaustion
Structure: Wholly owned
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Anti-CD83 autologous CAR-T
Description: Autologous CAR T cell therapy in development for AML and other oncology and autoimmune indications
Structure: Collaboration with Moffitt Cancer Center (CRISPR retains commercial rights)
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Anti-GPC3 autologous CAR-T
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)
Regenerative Medicine
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VCTX210: Type I diabetes mellitus
Description: Allogeneic, gene-edited, immune-evasive, stem cell-derived beta-cell replacement therapy
Structure: Collaboration with ViaCyte
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VCTX211: Type I diabetes mellitus
Description: Investigational allogeneic, gene-edited, immune-evasive, stem cell-derived beta-cell replacement therapy with additional gene edits that aim to further enhance cell fitness
Structure: Collaboration with ViaCyte
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VCTX212: Type I/II diabetes mellitus
Description: Investigational allogeneic, gene-edited, immune-evasive, stem cell-derived beta-cell replacement therapy with additional gene edits that aim to further enhance cell fitness
Structure: Collaboration with ViaCyte
In Vivo Approaches
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CTX310: ANGPTL3 for cardiovascular disease
Description: In vivo gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to reduce expression of ANGPTL3
Structure: Wholly owned
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CTX320: Lp(a) for cardiovascular disease
Description: In vivo investigational gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to reduce expression of Lp(a)
Structure: Wholly owned
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CTX330: PCSK9 for cardiovascular disease
Description: In vivo investigational gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to reduce expression of PCSK9
Structure: Wholly owned
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Hemophilia A
Description: In vivo investigational gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to insert a corrected factor VIII gene
Structure: Collaboration with Bayer
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Undisclosed deletion and insertion programs
Description: In vivo investigational gene-editing therapies delivered using lipid nanoparticles or adeno-associated viral vectors
Structure: Various
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Friedreich's ataxia (FA)
Description: In vivo gene-editing therapy delivered with an engineered adeno-associated virus (AAV) in development for the treatment of Friedreich’s ataxia
Structure: Collaboration with Capsida Biotherapeutics
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Amyotrophic lateral sclerosis (ALS)
Description: In vivo gene-editing therapy delivered with an engineered adeno-associated virus (AAV) in development for the treatment of familial ALS
Structure: Collaboration with Capsida Biotherapeutics
Download the CRISPR Therapeutics Corporate Presentation
CTX112™, CTX121™, CTX131™, CTX310™, CTX320™, CTX330™, VCTX210™, VCTX211™ and VCTX212™ are trademarks and registered trademarks of CRISPR Therapeutics AG.
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