Pipeline

Tackling a range of diseases with different approaches

We have established a portfolio of programs by selecting disease targets based on a number of criteria, including unmet medical need, technical feasibility, advantages of CRISPR/Cas9 relative to other approaches and time required to advance the product candidate into and through clinical trials.

Icon Hemo 80X80

Hemoglobinopathies

 
Research
Ind-Enabling
Clinical
Marketed
 
  • CTX001
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    Description: CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from β-thalassemia and sickle cell disease

    Gene editing approach: Disruption

    Ownership: Co-development and co-commercialization with Vertex

    For more information on CTX001 please click here

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Immuno-Oncology

 
Research
Ind-Enabling
Clinical
Marketed
 
  • CTX110
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    Description: CTX110 is an allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapy targeting CD19 in development for the treatment of CD19+ malignancies

    Gene editing approach: Disruption and insertion

    Ownership: 100% owned by CRISPR Therapeutics

    For more information on CTX110 please click here

  • CTX120
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    Description: CTX120 is an allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapy targeting BCMA in development for the treatment of multiple myeloma

    Gene editing approach: Disruption and insertion

    Ownership: 100% owned by CRISPR Therapeutics

    For more information on CTX120 please click here

  • CTX130
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    Description: CTX130 is an allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapy targeting CD70 in development for the treatment of both solid tumors and hematologic malignancies

    Gene editing approach: Disruption and insertion

    Ownership: 100% owned by CRISPR Therapeutics

    For more information on CTX130 please click here

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Regenerative Medicine

 
Research
Ind-Enabling
Clinical
Marketed
 
  • Diabetes mellitus type 1
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    Description: Allogeneic beta-cell replacement therapy derived from a CRISPR/Cas9 gene-edited immune-evasive stem cell line in development for the treatment of diabetes

    Gene editing approach: Disruption and insertion

    Ownership: Co-development and co-commercialization with ViaCyte

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In Vivo and Other Genetic Diseases

 
Research
Ind-Enabling
Clinical
Marketed
 
  • Glycogen storage disease type Ia (GSD Ia)
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    Description: In vivo CRISPR/Cas9-based candidate in development for patients suffering from GSD Ia, a rare genetic disease caused by mutations in the G6PC gene encoding glucose-6-phosphatase. Without glucose-6-phosphatase, the liver cannot break down glycogen, which leads to a variety of complications

    Gene editing approach: Correction/insertion

    Ownership: 100% owned by CRISPR Therapeutics

  • Duchenne muscular dystrophy (DMD)
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    Description: In vivo CRISPR/Cas9-based candidate in development for patients suffering from DMD, a rare, X-linked genetic disease caused by mutations in the dystrophin gene. The absence of dystrophin protein, which plays a key structural role in muscle fiber function, leads to muscle degeneration, loss of mobility and premature death in this disease

    Gene editing approach: Deletion

    Ownership: 100% owned by CRISPR Therapeutics

  • Cystic fibrosis (CF)
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    Description: In vivo CRISPR/Cas9-based candidate in development for patients suffering from CF, a rare genetic disease caused by mutations in the CFTR, or cystic fibrosis transmembrane conductance regulator, gene. Patients with CF develop thick mucus in vital organs, particularly the lungs, pancreas and gastrointestinal tract, leading to chronic severe respiratory infections, chronic lung inflammation, poor absorption of nutrients, progressive respiratory failure and early mortality

    Gene editing approach: Correction/insertion

    Ownership: License option with Vertex

  • Hurler syndrome (MPS I)
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    Description: Ex vivo autologous CRISPR/Cas9 gene-edited hematopoietic stem cell candidate in development for patients suffering from MPS I, a rare genetic disease caused by mutations in the IDUA gene encoding alpha-L-iduronidase. The absence of alpha-L-iduronidase results in the accumulation of glycosaminoglycans (GAGs) leading to cellular dysfunction and severe clinical abnormalities

    Gene editing approach: Correction/insertion

    Ownership: 100% owned by CRISPR Therapeutics

 

Learn more about our programs and approaches