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Cyclerion Therapeutics, Inc., a biopharmaceutical company, develops treatments for serious diseases. It is developing Olinciguat, an orally administered vascular soluble guanylate cyclase (sGC) stimulator, which is in Phase 2 clinical trial to out-license for cardiovascular diseases; and Praliciguat, a systemic sGC stimulator that is licensed to Akebia Therapeutics, Inc. for the treatment of rare kidney disease. The company was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.

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.

Larimar Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing treatments for rare diseases using its novel cell penetrating peptide technology platform. Its lead product candidate is CTI-1601, which is in Phase 2 OLE clinical trial for the treatment of Friedreich's ataxia, a rare, progressive and fatal genetic disease. Larimar Therapeutics, Inc. is based in Bala Cynwyd, Pennsylvania.

NextCure, Inc., a clinical-stage biopharmaceutical company, engages in discovering and developing novel immunomedicines to treat cancer and other immune-related diseases by restoring normal immune function. The company is developing NC410, an immunomedicine designed to block immune suppression mediated by LAIR-1; NC762, a monoclonal antibody that binds specifically to B7-H4, a protein expressed on multiple tumor types; and NC525, a monoclonal antibody that binds to LAIR-1 to functionally kill AML blast cells and leukemic stem cells while preserving normal blood cells, including hematopoietic stem cells. Its discovery and research programs include an antibody in preclinical evaluation of other potential novel immunomodulatory molecules. It has a license agreement with Yale University. The company was incorporated in 2015 and is headquartered in Beltsville, Maryland.