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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.

Gemini Therapeutics, Inc., a clinical stage precision medicine company, engages in developing various therapeutic compounds for treating genetically defined age-related macular degeneration (AMD). Its lead candidate is GEM103, a recombinant form of the human complement factor H protein to treat dry AMD patients. The company also develops GEM307 for treatment of systemic diseases. Gemini Therapeutics, Inc. was founded in 2015 and is headquartered in Cambridge, Massachusetts.

Immunic, Inc., a biotechnology company, develops a pipeline of selective oral immunology therapies for the treatment of chronic inflammatory and autoimmune diseases in the United States and Germany. Its lead development program is IMU-838, which is in Phase 3 clinical trial, for treatment of multiple sclerosis, including relapsing and progressive multiple sclerosis; and moderate-to-severe ulcerative colitis. The company is also developing IMU-856, which is entering Phase 2 clinical trial, for the restoration of the intestinal barrier function in patients suffering from gastrointestinal diseases, such as celiac disease, inflammatory bowel disease, short bowel syndrome, irritable bowel syndrome with diarrhea, and other intestinal barrier function diseases; and IMU-381, which is in preclinical trial, for the treatment of gastrointestinal diseases. Immunic, Inc. was founded in 2016 and is headquartered in New York, New York.

Orphazyme A/S, a biopharmaceutical company, develops therapies for the treatment of neurodegenerative orphan diseases. The company focuses on the amplification of heat-shock proteins to develop and commercialize therapeutics for diseases caused by protein misfolding and aggregation, and lysosomal dysfunction, including lysosomal storage and neuromuscular degenerative diseases. Its lead candidate is the Arimoclomol, which is in clinical development for four orphan diseases, including Niemann-Pick disease type C, Amyotrophic Lateral Sclerosis, Inclusion Body Myositis, and Gaucher disease. The company was incorporated in 2009 and is headquartered in Copenhagen, Denmark.

Xeris Biopharma Holdings, Inc., a biopharmaceutical company, engages in developing and commercializing therapies in Illinois. The company offers Gvoke, a ready-to-use liquid-stable glucagon for the treatment of severe hypoglycemia pediatric and adult patients; Keveyis, a therapy for the treatment of hyperkalemic, hypokalemic, and related variants of primary periodic paralysis; and Recorlev, a cortisol synthesis inhibitor proved for the treatment of endogenous hypercortisolemia in adult patients with Cushing's syndrome. It is also developing XP-8121, a once-weekly subcutaneous injection of levothyroxine that is in phase I clinical trial for the treatment of hypothyroidism; and non-aqueous XeriSol and XeriJect technologies for various therapies. The company was incorporated in 2005 and is headquartered in Chicago, Illinois.