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MeiraGTx Holdings PLC is a clinical-stage gene therapy company. It focuses on the eye, salivary gland, and central nervous system disorders. The company's pipeline products include AAV-CNGB3, AAV-CNGA3 and AAV-RPGR, and others. Geographically, it operates in the United States, the United Kingdom, and European Union.
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.