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CRISPR Therapeutics AG (CRSP) is a gene editing company.
The company focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9) gene-editing platform in Switzerland.
Its lead product candidate is CTX001, which targets sickle cell disease and beta-thalassemia with an ex vivo approach whereby cells are harvested from a patient, treated with a CRISPR/Cas9-based therapeutic and reintroduced into the patient.
The company is also developing CTX101, a donor-derived gene-edited allogeneic CAR-T therapy targeting CD19-positive malignancies.
In addition, it is developing earlier stage allogeneic CAR-T programs targeting B-Cell maturation antigen and CD70; hemoglobinopathies to treat other diseases, including Hurler Syndrome and severe combined immunodeficiency disease; programs that are in preclinical development for indications, including glycogen storage disease Ia and hemophilia; and programs targeting diseases of organ systems outside the liver, such as Duchenne muscular dystrophy and cystic fibrosis.
It has a collaboration agreements with Vertex Pharmaceuticals, Incorporated Vertex Pharmaceuticals (Europe) Limited to develop, manufacture, commercialize, sell, and use therapeutics; and StrideBio, Inc. to develop adeno-associated viral capsids with enhanced properties for in vivo gene editing programs.
The company also has research collaboration agreements with Neon Therapeutics for developing neoantigen-based therapeutic vaccines and T cell therapies; Massachusetts General Hospital Cancer Center to develop T cell therapies for cancer; ViaCyte, Inc. for designing allogeneic cell therapies derived from gene edited human stem cells for use in the treatment of diabetes type 1, diabetes type 2, and insulin dependent diabetes; and ProBioGen AG to develop novel in vivo delivery modalities for CRISPR/Cas9.
Shares rose last week after the companu and its partner Vertex Pharmaceuticals VRTX announced the dosing of the first patient in a phase I/II study evaluating the CRISPR/Cas9 gene-editing therapy, CTX001, in patients with beta thalassemia. The companies also enrolled first patients in another phase I/II study evaluating CTX001 in patients with severe sickle cell disease (“SCD”), a severe hereditary form of anemia. Dosing in the study is expected to start in mid-2019.
The company remains on track to initiate an immuno-oncology study in the first half of 2019 on its CAR-T cell therapy candidate, CTX110, for treating CD19+ malignancies. The company is the sole owner of the candidate. Since September, the company has inked or modified several collaboration agreements with other pharma companies for pre-clinical development of its new CRISPR/Cas9 gene editing candidates.
The news has sent shares into a bullish "cup and handle" formation are expected to move higher out of this formation.
Entry Point: $39.00
Stop Loss: $37.00
Trading Ranege: $22.22 - $73.90
Target Price: $42.90
CRSP closed at $35.30
