CRISPR Therapeutics (NASDAQ:CRSP) is a biotechnology company pioneering the development of transformative gene‐based medicines using CRISPR/Cas9 gene editing technology. Founded in 2013 and headquartered in Zug, Switzerland, with major research and development centers in Cambridge, Massachusetts, the company focuses on harnessing its proprietary CRISPR/Cas9 platform to precisely modify target genes implicated in serious diseases. By leveraging the programmability of CRISPR/Cas9, CRISPR Therapeutics seeks to create treatments that address the root cause of genetic disorders rather than solely managing symptoms.
The company’s leading product candidate, CTX001, is being developed in collaboration with Vertex Pharmaceuticals to treat sickle cell disease and transfusion‐dependent beta‐thalassemia by editing patients’ own hematopoietic stem cells. Beyond its hematology programs, CRISPR Therapeutics is advancing a diversified pipeline spanning oncology, regenerative medicine and rare diseases. Key oncology programs include CTX110, an allogeneic CAR-T cell therapy targeting CD19, and CTX120, an allogeneic CAR-T cell therapy directed at BCMA for multiple myeloma. The company also pursues in vivo gene editing candidates for conditions such as diabetes and muscular dystrophy.
Since its inception, CRISPR Therapeutics has forged collaborations with leading research institutions and pharmaceutical companies to accelerate both preclinical and clinical development. In addition to its partnership with Vertex, the company has established research agreements with institutions such as the University of California, Berkeley and the German Cancer Research Center. These alliances enable CRISPR Therapeutics to combine its gene‐editing expertise with complementary scientific and clinical capabilities, advancing programs through regulatory milestones and clinical trials.
Under the leadership of President and Chief Executive Officer Samarth Kulkarni, CRISPR Therapeutics employs a global team of scientists, clinicians and regulatory experts. The company maintains operations across North America and Europe, supporting its mission to deliver groundbreaking therapies to patients worldwide. By integrating cutting‐edge science with rigorous clinical development and commercial planning, CRISPR Therapeutics aims to establish a new paradigm in medicine—one where precise genomic corrections provide durable and potentially curative treatments for diseases with high unmet medical need.
