Depending on the therapeutic application, these technologies can be deployed either on human cells outside the body (ex vivo) or directly on cells and tissues inside the body (in vivo).
To effectively bring zinc finger technologies into targeted cells, we employ delivery vectors, which serve as vehicles for transporting genetic material. These vectors can originate from inactivated viruses, such as adeno-associated virus (AAV) or lentivirus (LVV).
Committed to refining zinc finger technologies, our goal is to offer effective and safe therapeutic options that address the needs of patients with diverse diseases. Our considerable experience using AAVs in clinical trials supports our belief in the safety of AAVs for delivering genomic medicines. To date, more than 200 patients across our various clinical trials and therapeutic areas have been dosed with AAV therapies that have been generally well tolerated.
Learn more about our zinc finger platform
.