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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.

Clementia Pharmaceuticals Inc., a clinical stage biopharmaceutical company, develops treatments for patients suffering from bone disorders and other diseases. Its lead product candidate is palovarotene, an oral small molecule for the treatment of fibrodysplasia ossificans progressive, multiple osteochondromas, dry eye disease, and other diseases is in the Phase 3 MOVE Trial. Clementia Pharmaceuticals Inc. was founded in 2010 and is based in Montreal, Canada.

Enanta Pharmaceuticals, Inc., a biotechnology company, discovers and develops small molecule drugs for the treatment of viral infections and liver diseases. Its product pipeline comprises EDP-514, which is in phase 1b clinical development for the treatment of chronic infection with hepatitis B virus or HBV; EDP-938 and EDP-323, which is in phase II clinical development for the treatment of respiratory syncytial virus; EDP-235, which is in phase II clinical development for the treatment of human coronaviruses; and Glecaprevir, which is in the market for the treatment of chronic infection with hepatitis C virus or HCV. The company has a collaborative development and license agreement with Abbott Laboratories to develop, manufacture, and commercialize HCV NS3 and NS3/4A protease inhibitor compounds, including paritaprevir and glecaprevir. Enanta Pharmaceuticals, Inc. was incorporated in 1995 and is headquartered in Watertown, Massachusetts.

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.

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.