CRISPR Therapeutics develops gene-based medicines using CRISPR/Cas9 gene-editing technology to treat serious human diseases. Founded in 2013, the company has progressed from research stage to commercialisation, with operations spanning Switzerland, Boston and San Francisco. The company's proprietary SyNTase™ editing platform aims to enable precise and scalable gene correction for next-generation therapeutics.
CASGEVY™ (exagamglogene autotemcel), developed in collaboration with Vertex Pharmaceuticals, represents the company's flagship achievement - the world's first CRISPR-based gene-edited therapy approved for eligible patients with sickle cell disease or transfusion-dependent beta thalassemia. The therapy has received regulatory approval in several countries, marking a significant milestone in translating gene-editing science into clinical medicine.
The company's pipeline spans hemoglobinopathies, oncology, regenerative medicine, cardiovascular disease, autoimmune conditions and rare diseases. CRISPR Therapeutics operates state-of-the-art manufacturing facilities and emphasises strategic partnerships with established biopharmaceutical companies. The organisation traces its scientific foundations to the 2012 CRISPR/Cas9 research by Emmanuelle Charpentier and Jennifer Doudna, whose later Nobel Prize in Chemistry underscores the foundational importance of the technology the company builds upon.
