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Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need in the United States. The company's lead product candidate is AT-007 (also called govorestat) that has completed phase 3 for the treatment of galactosemia in healthy volunteers and adults, in pediatric clinical study for the treatment of galactosemia in kids, for treating enzyme sorbitol dehydrogenase, and for the treatment of phosphomannomutase enzyme-CDG. It also develops AT-001 (also called caficrestat) that is in phase 3 clinical trials to treat diabetic cardiomyopathy, as well as for the treatment of diabetic peripheral neuropathy; and AT-003, which is in preclinical studies for the treatment diabetic retinopathy. The company has exclusive license and supply agreement with Mercury Pharma Group Limited to commercialize drug products containing AT-007. Applied Therapeutics, Inc. was incorporated in 2016 and is headquartered in New York, New York.

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.

KalVista Pharmaceuticals, Inc., a clinical stage pharmaceutical company, engages in the discovery, development, and commercialization of small molecule protease inhibitors for diseases with unmet needs in the United States. The company's product portfolio comprises small molecule plasma kallikrein inhibitors targeting hereditary angioedema (HAE) and diabetic macular edema (DME); and oral plasma kallikrein inhibitors. Its products include sebetralstat, which is initiation of the Phase 3 KONFIDENT trial as a potential oral, on-demand therapy for HAE attacks; and Factor XIIa, an oral inhibitor program which is in preclinical stage targets an enzyme in HAE. The company is headquartered in Cambridge, Massachusetts.

Oculis Holding AG, a clinical-stage biopharmaceutical company, develops drug candidates to treat ophthalmic diseases. The company's lead product candidate is OCS-01, a topical dexamethasone optireach formulation, which is in Phase 3 clinical trials for the treatment of diabetic macular edema; OCS-02, a topical biologic candidate that is in Phase 2b clinical trials for the treatment for dry eye disease; and OCS-05, a disease modifying neuroprotective agent for neurological damage with indications for glaucoma, dry age-related macular degeneration and diabetic retinopathy, and acute optic neuritis. The company is based in Zug, Switzerland.