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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 the 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.
Zevra Therapeutics Inc is a rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. The company's mission is to bring life-changing therapeutics to people living with rare diseases. With data-driven development and commercialization strategies, it is overcoming complex drug development challenges to make new therapies available to the rare disease community.