Gene Replacement

Gene replacement is the most common type of gene therapy. To replace a mutated or missing gene, a healthy copy of the gene is packaged into a viral vector that is optimized to enter the affected cells or organs. Once the healthy gene copy is delivered to the nucleus of the cell, the gene will continuously produce functional protein by the patient’s own cellular machinery. Alcyone utilizes Adeno Associated Virus 9 (AAV9), a viral vector that has been well characterized for safety and its ability to deliver genes to cells in the nervous system.