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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CD117 ADC / In vivo HSC editing
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 & Autoimmune Disease
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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-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)
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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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Other CAR T Programs
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 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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CTX340: AGT for refractory hypertension
Description: In vivo investigational gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to reduce expression of hepatic angiotensinogen (AGT).
Structure: Wholly owned
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CTX450: ALAS1 for acute hepatic porphyria (AHP)
Description: In vivo investigational gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to reduce expression of 5’-aminolevulinate synthase 1 (ALAS1).
Structure: Wholly owned
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Undisclosed Rare Disease Programs
Description: Undisclosed investigational in vivo gene-editing therapies for rare diseases
Structure: Wholly owned
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Undisclosed Common Disease Programs
Description: Undisclosed investigational in vivo gene-editing therapies for common diseases
Structure: Wholly owned
Regenerative Medicine
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CTX211: Device approach for Type 1 diabetes mellitus
Description: Investigational allogeneic, gene-edited, immune-evasive, stem cell-derived beta-cell replacement therapy
Structure: Wholly owned
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CTX213: Deviceless approach for Type 1 diabetes mellitus
Description: Unencapsulated investigational allogeneic, gene-edited, immune-evasive, stem cell-derived beta-cell replacement therapy
Structure: Wholly owned
Other Disclosed Partnered Programs
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Duchene’s Muscular Dystrophy (DMD)
Description: Investigational in vivo investigational gene-editing therapy for DMD
Structure: Licensed to Vertex
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Myotonic Dystrophy Type 1 (DM1)
Description: Investigational in vivo gene-editing therapy for DM1
Structure: Collaboration with Vertex
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Type 1 Diabetes Mellitus
Description: Gene-edited, hypoimmune islet cell therapies for T1D
Structure: Licensed to Vertex
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Cystic Fibrosis (CF)
Description: Investigational in vivo gene-editing therapy for CF
Structure: Licensed to Vertex
Download the CRISPR Therapeutics Corporate Presentation
CTX112™, CTX131™, CTX211™, CTX213™, CTX310™, CTX320™, CTX340™ and CTX450™ are trademarks and registered trademarks of CRISPR Therapeutics AG.
