Pipeline
Hemoglobinopathies
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Exa-cel: β-thalassemia
Partner: Vertex
Structure: Collaboration
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Exa-cel: Sickle cell disease (SCD)
Partner: Vertex
Structure: Collaboration
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Next-generation conditioning
Structure: Wholly-owned
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In vivo editing of HSCs
Structure: Wholly-owned
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
Structure: Wholly-owned
For more information on CTX110 please click here
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CTX112
Structure: Wholly-owned
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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
Structure: Wholly-owned
For more information on CTX130 please click here
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CTX131
Structure: Wholly-owned
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Anti-CD70 allogeneic CAR-NK
Partner: Nkarta Therapeutics
Structure: Collaboration
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CTX121: Anti-BCMA allogeneic CAR-T
Structure: Wholly-owned
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Anti-CD83 autologous CAR-T
Partner: Moffitt Cancer Center
Structure: Collaboration
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Anti-GPC3 autologous CAR-T
Partner: Roswell Park Comprehensive Cancer Center
Structure: Collaboration
Regenerative Medicine
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VCTX210: 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
Partner: ViaCyte
Structure: Collaboration
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VCTX211: Type I diabetes mellitus
Partner: ViaCyte
Structure: Collaboration
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VCTX212: Type I/II diabetes mellitus
Partner: ViaCyte
Structure: Collaboration
In Vivo Approaches
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CTX310: ANGPTL3
Structure: Wholly-owned
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CTX320: Lp(a)
Structure: Wholly-owned
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CTX330: PCSK9
Structure: Wholly-owned
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Hemophilia A
Partner: Bayer
Structure: Collaboration
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Undisclosed deletion and insertion programs
Structure: Various
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Friedreich's ataxia (FA)
Partner: Capsida Biotherapeutics
Structure: Collaboration
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Amyotrophic lateral sclerosis (ALS)
Partner: Capsida Biotherapeutics
Structure: Collaboration
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.
