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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.
Rapport Therapeutics Inc is a clinical-stage biopharmaceutical company focused on discovery and development of transformational small-molecule medicines for patients suffering from central nervous system disorders. Its foundational science has elucidated the complexities of neuronal receptor biology and enables the company to map and target certain neuronal receptor complexes. Neuronal receptors are complex assemblies of proteins, comprising receptor principal subunits and their receptor-associated proteins (RAPs), the latter of which play crucial roles in regulating receptor expression and function.