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Benitec Biopharma Inc is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines. Its technology has the potential to provide a one-shot cure for a wide range of diseases that are addressed by strict ongoing treatment regimens or that have no effective treatment or only palliative care options. The Company is developing a silence and replace-based therapeutic (BB-301) for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), a chronic, life threatening genetic disorder.
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.