NysnoBio is advancing a gene therapy for Parkinson's Disease based on human genetic data indicating the Parkin protein is a regenerative factor for neurons that die in Parkinson's Disease (PD). Persons born without the Parkin gene are diagnosed with PD typically by the time they are 25 years old and face a lifetime fo disability, due to the profound loss of neuronal function in the dopamine circuit. An abundance of published data demonstrates that adding Parkin protein to cells and animals can restore dopamine neurons to health after insults related to Parkinson's disease, supporting the use of Parkin therapies not only for Parkin-PD patients, but for all Parkinson's patients to restore health to dopamine neurons dying in PD. Our group has been developing therapies directed towards the Parkin protein for over 20 years and is comprised of the world's experts in Parkin biology and therapeutics. Our approach is straightforward and pragmatic. We have generated a gene therapy that is precisely delivered to the disease area and provides Parkin protein activity, allowing dopamine neurons to recover from disease and regain their normal function to produce dopamine to control normal movement. This groundbreaking therapy, solidly rooted in human genetics and human pathology is now poised to enter human trials within the next two years. In our CIRM program, we have advanced our Parkin gene therapy through pre-clinical cellular and animal model testing resulting in a Pre-IND meeting with the FDA that generated a road map for successful IND submission. The feedback from the FDA on our program was overwhelmingly positive, demonstrating that the data generated in the CIRM program was of high quality and specifically focused on moving this therapy to human patients as quickly, safely and efficiently as possible. Our plan is to test this therapy in genetic Parkin-PD patients first because this will be the most efficient way to generate positive clinical data in a small population. Because all PD patients can benefit from restoration of dopamine neurons, once we have clinical data on restoration of normal movement in Parkin-PD patients after Parkin gene therapy, we will test our therapy in additional sporadic and genetic PD patient populations. Our goal is to provide a transformative regenerative therapy to address the growing population of PD patients in California and around the world.
