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Savara Inc is a biopharmaceutical company. It is focused on rare respiratory diseases. The company's program, molgramostim nebulizer solution (molgramostim), is an inhaled granulocyte-macrophage colony-stimulating factor in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). It has one segment specialty pharmaceuticals within the respiratory system.
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.