The group's principle activity is to develop novel therapeutics based on ribonucleic acid interference. The group also develops an RNAi therapeutic for the treatment of hypercholesterolemia. The group provides treatments for diseases including Parkinson’s disease (PD), Huntington’s disease (HD), neuropathic pain, progressive multifocal leukoencephalopathy (PML), Ebola virus infection and cystic fibrosis (CF). The group operates from United States.
