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 ↗Sana Biotechnology, Inc. Common Stock
Sana Biotechnology, Inc., a biotechnology company, focuses on utilizing engineered cells as medicines in the United States. It develops ex vivo and in vivo cell engineering platforms for various therapeutic areas with unmet treatment needs, including type 1 diabetes, B cell mediated autoimmune diseases, and oncology. The company's product pipeline consist of UP421, a HIP-modified allogeneic primary islet cell product, which is in phase 1 clinical trial to treat type 1 diabetes; SC451, a HIP-modified, stem cell derived pancreatic islet cell therapy, which is in preclinical trial for the treatment of type 1 diabetes; SC291, a HIP-modified allogeneic CD19-dirtected allogeneic CAR T cell product candidate which is in phase 1 clinical trial to treat systemic lupus erythematosus and ANCA-associated vasculitis, as well as in preclinical trial to treat other indications and b-cell mediated autoimmune disease; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. It also develops SC262, a hypoimmune-modified CD22-directed allogeneic CAR T program, which is in phase 1 to treat non-Hodgkin's lymphoma; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. The company has an option and license agreement with Beam Therapeutics Inc. for use of Beam's proprietary CRISPR Cas12b nuclease editing technology to research, develop, and commercialize engineered cell therapy products; and a license agreement with Harvard College to access certain intellectual property for the development of hypoimmune-modified cells. The company was formerly known as FD Therapeutics, Inc. and changed its name to Sana Biotechnology, Inc. in September 2018. Sana Biotechnology, Inc. was incorporated in 2018 and is headquartered in Seattle, Washington.
Open SANA dashboard ↗