PIPELINE

LIVER DISEASE

Our liver disease pipeline is focused on developing gene therapies for a range of liver disorders, including genetic liver diseases and liver cancer. Our lead program is a gene therapy for the treatment of Polycystic liver disease (PCLD) which is an inherited disorder caused by mutations in the PKD1 or PKD2 genes. Mutations in these genes can lead to the growth of multiple cysts in the liver, which can cause liver enlargement, pain, and other symptoms. We are also developing gene therapies for other genetic liver diseases, such as hemophilia and Wilson disease. We believe that our liver disease pipeline has the potential to make a significant impact on the lives of patients suffering from these devastating diseases, and we are committed to advancing these programs as quickly and safely as possible.

CANCER

Our cancer therapy program using the suicide gene is focused on developing a gene therapy approach for the treatment of certain types of cancer, such as prostate cancer and glioblastoma multiforme. Our approach involves delivering a gene encoding a "suicide" enzyme into cancer cells, which then convert a prodrug into a toxic compound that kills the cancer cells. Our approach has several advantages over traditional cancer treatments, such as chemotherapy and radiation therapy, which can also damage healthy cells and tissues. By selectively targeting cancer cells, our therapy has the potential to be more effective and less toxic to the patient. We are currently conducting preclinical studies to evaluate the safety and efficacy of our suicide gene therapy approach. We believe that our approach has the potential to be a game-changer in the field of cancer therapy, and we are excited to continue our research in this area.

OCULAR DISEASE

Our ocular disease pipeline is focused on developing gene therapies for a range of eye disorders, including inherited retinal diseases and age-related macular degeneration. Our lead program is a gene therapy for the treatment of Leber congenital amaurosis (LCA), a rare genetic disorder that can lead to progressive vision loss and blindness. In addition to our LCA program, we are also developing gene therapies for other inherited retinal diseases, such as retinitis pigmentosa and Stargardt disease. Our approach involves delivering a functional copy of the defective gene to the affected cells in the eye, with the aim of restoring vision and preventing further damage. We are also developing gene therapies for age-related macular degeneration (AMD), a leading cause of vision loss in older adults. We believe that our ocular disease pipeline has the potential to transform the lives of patients suffering from these debilitating conditions.

DEGENERATIVE DISEASE

Our degenerative disease pipeline is focused on developing gene therapies for the treatment of debilitating conditions such as Duchenne muscular dystrophy, Huntington's disease, and amyotrophic lateral sclerosis (ALS). Our lead program is focused on developing a gene therapy for Duchenne muscular dystrophy, a progressive muscle fiber degeneration and weakness. Our approach involves delivering a gene encoding a protein that promotes the survival and function of these muscle, with the goal of slowing or halting the progression of the disease. In addition to DMD we are also developing gene therapies for Huntington's disease and ALS, both of which are characterized by the progressive degeneration of nerve cells in the brain and spinal cord. Our approach involves can correct the underlying genetic mutations that cause these diseases, with the goal of halting disease progression and improving patient outcomes.