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.
Hemoglobinopathies
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CTX001
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
Immuno-Oncology
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CTX110
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
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CTX120
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
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CTX130
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
Regenerative Medicine
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Type I diabetes mellitus
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
In Vivo Approaches
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Glycogen storage disease type Ia (GSD Ia)
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
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Duchenne muscular dystrophy (DMD)
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: Various
Ownership: Exclusive license to Vertex
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Myotonic dystrophy type 1 (DM1)
Description: In vivo CRISPR/Cas9-based candidate in development for patients suffering from DM1, a rare genetic disease caused by a trinucleotide repeat expansion in the DMPK gene. Patients with congenital DM1 have severe generalized weakness at birth (hypotonia), often causing complications with breathing and early death
Gene editing approach: Various
Ownership: Collaboration, option to co-develop and co-commercialize with Vertex
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Cystic fibrosis (CF)
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
Learn more about our programs and approaches
