Pipeline

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

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

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

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

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

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

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

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

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