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Regenxbio Inc is a biotechnology company. Its main activity is the development and commercialization of recombinant adeno associated virus gene therapy to correct an underlying genetic defect. The diseases that Regenxbio platform targets are metabolic (homozygous familial hypercholesterolemia), neurodegenerative conditions (mucopolysaccharidosis), and retinal diseases (wet age-related macular degeneration, X-linked retinitis pigmentosa). The company derives the majority of its revenue from the United States.
Anavex Life Sciences Corp is a clinical-stage biopharmaceutical company engaged in the development of differentiated therapeutics by applying precision medicine to central nervous system diseases with high unmet need. It analyzes genomic data from clinical studies to identify biomarkers, which are used to select patients that will receive the therapeutic benefit for the treatment of neurodegenerative and neurodevelopmental diseases. Its necessary compound ANAVEX 2-73 is being developed to treat Alzheimer's disease, Parkinson's disease, and potentially other central nervous system diseases, including rare diseases, such as Rett syndrome, a rare severe neurological monogenic disorder. The company's operating segment is into developing novel therapies for the management of CNS diseases.