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MIRM vs NTLA

Mirum Pharmaceuticals, Inc. Common Stock and Intellia Therapeutics, Inc

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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.

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Intellia Therapeutics, Inc

Intellia Therapeutics, Inc., a clinical-stage gene editing company, focuses on the development of curative genome editing treatments. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; and NTLA-2002 that is in Phase 1/2 study for the treatment of hereditary angioedema. It also focusses on the research of proprietary programs focused on developing engineered cell therapies to treat various cancers and autoimmune diseases. The company has license and collaboration agreement with AvenCell Therapeutics, Inc. to develop allogeneic universal CAR-T cell therapies; Kyverna Therapeutics, Inc. for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of various of B cell-mediated autoimmune diseases; ONK Therapeutics, Ltd. for the development of engineered NK cell therapies to cure patients with cancer; and ReCode Therapeutics, Inc. to develop novel genomic medicines for the treatment of cystic fibrosis. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.

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