Develops CRISPR/Cas9 gene editing technology for therapeutic applications in genetic disorders and cancer.
CRISPR Therapeutics AG is a pioneering gene editing company headquartered in Zug, Switzerland, focused on advancing gene-based therapies for serious diseases using its proprietary CRISPR/Cas9 platform. This innovative technology enables precise modifications to genomic DNA, offering potential treatments across a broad spectrum of medical conditions.
The company's robust portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Among its leading initiatives is CTX001, an ex vivo CRISPR gene-edited therapy designed to treat transfusion-dependent beta-thalassemia and severe sickle cell disease. This groundbreaking approach involves engineering a patient's hematopoietic stem cells to produce elevated levels of fetal hemoglobin in red blood cells, potentially providing a transformative treatment option.
CRISPR Therapeutics is also advancing other promising candidates such as CTX110, CTX120, and CTX130, which are gene-edited allogeneic CAR-T therapies targeting various malignancies. Additionally, the company is developing VCTX210, a gene-edited immune-evasive stem cell-derived therapy for type 1 diabetes, and exploring in vivo gene-editing programs targeting diseases of the liver, lung, muscle, and central nervous system. With strategic partnerships with industry leaders like Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and others, CRISPR Therapeutics remains at the forefront of innovation in gene editing, striving to deliver impactful therapies that address unmet medical needs globally.