> Our Mission
REIMAGINING THE FUTURE FOR PEOPLE LIVING WITH NEUROLOGICAL DISORDERS
We strive to improve the lives of individuals and families living with Parkinson's disease and other neurological disorders through collaborative scientific, medical, and healthcare advancements that deliver safe and efficacious gene therapies for slowing or reversing disease progression.
> Our Focus
GENE THERAPY FOR PARKINSON'S DISEASE
Brain Neurotherapy Bio is focused on the clinical translation and development of neurotrophic factor gene therapy for the treatment of Parkinson’s disease and other neurological conditions. Our initial clinical program will expand upon the safety and evaluate the preliminary efficacy of glial cell line-derived neurotrophic factor (GDNF) gene therapy in patients with Parkinson’s disease. This program utilizes a clinically-proven AAV gene therapy platform that incorporates novel image-guided neurosurgical delivery technology to optimize the precision, safety and efficiency of gene transfer within the brain. Our vision is to provide people afflicted with neurological disorders with increased access to transformative gene therapies for slowing or reversing disease progression.
> Our Science
ADVANCING BRAIN-TARGETED GENE THERAPIES
Gene therapy directly administered to the brain features the delivery of an engineered gene packaged within a carrier particle, that allows targeting and influence of specific populations of brain cells. We take advantage of optimized AAV gene transfer technology and advanced neurosurgical methods to precisely deliver therapeutic genes within the brain. Following incorporation of the desired DNA sequences, or genes, within targeted brain cells, the DNA harnesses intrinsic cell machinery to produce the therapeutic product. In the Parkinson's disease study, this product would be GDNF that is used by targeted brain cells to enhance function and maintain viability of specific brain cells and their associated networks.