Mirum Pharmaceuticals, Inc. Common Stock
Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.
Open MIRM dashboard ↗Ocugen, Inc. Common Stock
Ocugen, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel gene and cell therapies, biologic, and vaccines that improve patients' health. The company's pipeline product includes OCU400, a novel gene therapy product candidate restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases, currently under Phase 3 trials for the treatment of retinitis pigmentosa and Phase 1/2 trials for the treatment of leber congenital amaurosis; OCU410, a gene therapy under phase 1/2 for the treatment of dry age-related macular degeneration (AMD); and OCU410ST, a gene therapy under phase 1/2 for the treatment of Stargardt disease. It is involved in the development of OCU200, a novel fusion protein for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration; and NeoCart, an autologous chondrocyte-derived neocartilage, currently under Phase 3 studies indicated for the repair of knee cartilage injuries in adult. In addition, the company is developing OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. It has collaboration agreements with National Institute of Allergy and Infectious Diseases for early clinical studies for the OCU500 program; and a strategic partnership with CanSino Biologics Inc. for manufacturing its modifier gene therapy pipeline product candidates. The company was founded in 2013 and is headquartered in Malvern, Pennsylvania.
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