We are a genome editing company that began operations in 2015 to develop medicines to deliver the potential benefits of genetic medicine in the fight against rare diseases in pediatric patients. Our scientific co-founders — Mark Kay, M.D., Ph.D., Adi Barzel, Ph.D., and Leszek Lisowski, Ph.D., MBA — are global leaders in gene therapy and gene editing. It was their research at Stanford University that led to the development of our foundational technology platform, GeneRide.
GeneRide is designed to precisely and stably integrate corrective genes into a patient’s genome to provide a durable therapeutic benefit from a single treatment. Our technology harnesses a natural DNA repair process called homologous recombination, which we believe will enable us to insert therapeutic genes into specific locations in the chromosome without the use of exogenous nucleases (“molecular scissors” that are engineered to cut DNA), which are used in other gene editing technologies. Our approach is designed to offer a safe and precise way to intervene and help patients with severe genetic disease.
We are initially targeting rare liver disorders where it is critically important to treat a patient early in life. A drawback of traditional gene therapy approaches in treating such disorders is that their effects are diluted as the patient’s liver cells divide and the liver grows. By contrast, GeneRide has the potential to promote a stable and durable change that persists in the patient’s cells as they divide. After evaluating our therapy in liver disorders, we intend to deploy additional potential therapies based on GeneRide in other tissues.
Our team is headquartered in Cambridge, Mass., where we are working with a shared sense of urgency to develop the next generation of genetic medicines.