With close ties to University of North Carolina, Durhma, Neurogt is structured around developing gene therapy products to treat rare neurogenetic diseases. The major research focus of the principals has been development of effective gene delivery targeting the entire nervous system, using adeno-associated virus (AAV) vectors, for treating neuropathic lysosomal storage diseases in humans. Research also has targeted the critical challenge in translating rAAV gene therapy to clinical application, the widespread pre-existing antibodies against AAV in humans. Principals have demonstrated that effective antibody depletion requires broad immunomodulation and are testing a novel transient antibody clearance approach for AAV gene delivery. Further, the blood-brain-barrier (BBB) has been a major obstacle in therapeutic development for neurological diseases. To address this challenge, we are working towards develop novel techniques facilitating/enhancing AAV gene delivery to the central nervous system (CNS) across the BBB.
