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Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

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.

Protagonist Therapeutics, Inc., a biopharmaceutical company, develops peptide-based drugs for hematology and blood disorders, and inflammatory and immunomodulatory diseases. It is developing Rusfertide (PTG-300), an injectable hepcidin mimetic that completed phase 2 clinical trials for the treatment of polycythemia vera and other blood disorders; and JNJ-2113, an orally delivered investigational drug to block biological pathways that completed phase 2b clinical trials for the treatment of moderate-to-severe plaque psoriasis; and PN-943, an orally delivered, gut-restricted alpha 4 beta 7 specific integrin antagonist completed a phase 2 clinical trials in patients with moderate to severe ulcerative colitis. The company has a license and collaboration agreement with Takeda to commercialize rusfertide; and JNJ Innovative Medicines to co-develop Interleukin-23 receptor antagonist compound JNJ-2113. Protagonist Therapeutics, Inc. was incorporated in 2006 and is headquartered in Newark, California.

RAPT Therapeutics, Inc., a clinical-stage immunology-based biopharmaceutical company, focuses on discovery, development, and commercialization of oral small molecule therapies for patients with unmet needs in oncology and inflammatory diseases in the United States. The company's lead inflammation drug candidate is zelnecirnon (RPT193), a C-C motif chemokine receptor 4 (CCR4) antagonist that selectively inhibit the migration of type 2 T helper cells into inflamed tissues. Its lead oncology drug candidate is tivumecirnon (FLX475), an oral small molecule CCR4 antagonist that is in the Phase 1/2 clinical trial to investigate as a monotherapy and in combination with pembrolizumab in patients with advanced cancer. The company was formerly known as FLX Bio, Inc. and changed its name to RAPT Therapeutics, Inc. in May 2019. RAPT Therapeutics, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California. RAPT Therapeutics, Inc. operates as a subsidiary of Bristol-Myers Squibb Company

Syros Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of treatment for hematologic malignancies. The company's lead product candidates are Tamibarotene, a selective retinoic acid receptor alpha agonist, which is in Phase III clinical trial for genomically defined subset of patients with myelodysplastic syndrome and Phase II clinical trial for patients with acute myeloid leukemia; SY-2101, a novel oral form of arsenic trioxide for treating patients with acute promyelocytic leukemia; and SY-5609, a cyclin-dependent kinase 7 inhibitor, which is in a Phase I clinical trial in patients with select advanced solid tumors. The company was formerly known as LS22, Inc. and changed its name to Syros Pharmaceuticals, Inc. in August 2012. Syros Pharmaceuticals, Inc. was incorporated in 2011 and is headquartered in Cambridge, Massachusetts.