Maire Osborn, PhD, Associate Director, Neuro Discovery, Dicerna Pharmaceuticals
Dicerna’s GalXC RNAi technology provides safe and durable treatment for genetically defined diseases. Recent efforts have focused on GalXC-Plus, a suite of novel RNAi platforms enabling extrahepatic delivery to the central nervous system (CNS), muscle, adipose, and adrenal tissues. The biggest hurdle to developing GalXC-Plus for neurological disease is the need for efficient delivery to specific neural cell types. We will discuss engineering challenges and advances enabling long-lasting, cell-specific gene silencing in the brain, spinal cord, and peripheral nerves of rodents and non-human primates. These discoveries position GalXC-Plus to be effective against a broad range of CNS disorders with no available treatment options.