Editas Medicine (NASDAQ:EDIT) is a biotechnology company focused on the development of genome editing therapies to treat a range of serious diseases. Founded in 2013 and headquartered in Cambridge, Massachusetts, the company harnesses CRISPR/Cas9 and CRISPR/Cas12a (Cpf1) gene-editing platforms to design precision medicines that directly target the underlying genetic causes of disease. Editas Medicine’s vision centers on advancing in vivo and ex vivo gene editing solutions for patients with inherited retinal disorders, hematologic conditions, and other rare diseases with high unmet medical need.
The company’s pipeline includes multiple product candidates in various stages of clinical and preclinical development. The most advanced program, EDIT-101, is designed to treat Leber congenital amaurosis type 10 (LCA10), a hereditary retinal disorder that causes early-onset vision loss. In addition, Editas is advancing ex vivo cell therapies such as EDIT-301 for sickle cell disease and beta-thalassemia, utilizing engineered hematopoietic stem cells to correct pathogenic mutations. Earlier-stage research leverages proprietary delivery technologies and novel editor variants to expand the reach of gene editing beyond the eye and blood, positioning the company to address additional genetic and ocular diseases.
Since its inception, Editas Medicine has built partnerships with leading pharmaceutical and academic institutions across North America, Europe and Asia to accelerate research and development. The company maintains research collaborations with global organizations to refine delivery vectors, optimize editing efficiency, and ensure the safety of its therapeutic candidates. These alliances amplify Editas Medicine’s capabilities in translational science while facilitating ultimate patient access through potential co-development and commercialization pathways.
Leadership at Editas Medicine combines scientific expertise with biopharmaceutical experience. The executive team, led by President and Chief Executive Officer Gilmore O’Neill, includes seasoned professionals in gene editing, clinical development and regulatory affairs. The company’s board and scientific advisory committees feature pioneers in molecular biology and genetics, guiding strategic decisions as Editas Medicine works toward its goal of bringing transformative, precisely targeted treatments to patients worldwide.
