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Generation Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Heron Therapeutics, Inc., a commercial-stage biotechnology company, focuses on enhancing the lives of patients by developing and commercializing therapeutic that enhances medical care. The company's product candidates utilize its proprietary Biochronomer, a drug delivery technology, which delivers therapeutic levels of a range of short-acting pharmacological agents over a period from days to weeks with a single administration. It offers SUSTOL (granisetron), an extended-release injection for the prevention of acute and delayed nausea and vomiting associated with moderately emetogenic chemotherapy, or anthracycline and cyclophosphamide combination chemotherapy regimens; and CINVANTI, an intravenous formulation of aprepitant, a substance P/neurokinin-1 receptor antagonist for the prevention of acute and delayed nausea and vomiting associated with highly emetogenic cancer chemotherapy, as well as nausea and vomiting associated with moderately emetogenic cancer chemotherapy. The company is also developing ZYNRELEF, a dual-acting local anesthetic that delivers a fixed-dose combination of the local anesthetic bupivacaine and a low dose of the nonsteroidal anti-inflammatory drug meloxicam; and APONVIE, an intravenous formulation of a substance P/neurokinin-1 receptor antagonist indicated for the prevention of postoperative nausea and vomiting in adults. The company was formerly known as A.P. Pharma, Inc. and changed its name to Heron Therapeutics, Inc. in January 2014. Heron Therapeutics, Inc. was founded in 1983 and is headquartered in San Diego, California.

Intercept Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics to treat progressive non-viral liver diseases in the United States, Europe, and Canada. The company markets Ocaliva, a farnesoid X receptor agonist used for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid in adults. It is also developing Ocaliva for various indications, including nonalcoholic steatohepatitis and NASH; and other product candidates in various stages of clinical and preclinical development. The company has a license agreement with Aralez Pharmaceuticals Canada Inc. to develop and commercialize bezafibrate in the United States. It markets its products through an internal commercial organization and third-party distributors. The company was incorporated in 2002 and is headquartered in Morristown, New Jersey. As of November 8, 2023, Intercept Pharmaceuticals, Inc. operates as a subsidiary of Alfasigma S.p.A..

MacroGenics, Inc., a biopharmaceutical company, develops, manufactures, and commercializes antibody-based therapeutics to treat cancer in the United States. Its approved product is MARGENZA (margetuximab-cmkb), a human epidermal growth factor receptor 2 (HER2) receptor antagonist indicated, in combination with chemotherapy, for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens. The company's pipeline of immuno-oncology product candidates includes MGC018, an antibody drug conjugate (ADC), which targets solid tumors expressing B7-H3; Enoblituzumab, a monoclonal antibody that targets B7-H3; and MGD024, an investigational bispecific CD123 × CD3 DART molecule to minimize cytokine-release syndrome for patients with hematologic malignancies. In addition, it develops Lorigerlimab, a monoclonal antibody that targets the immune checkpoints PD-1 and cytotoxic T-lymphocyte-associated protein 4; Tebotelimab, an investigational tetravalent DART molecule for PD-1 and lymphocyte-activation gene 3; Retifanlimab, a humanized monoclonal antibody targeting programmed death receptor-1; and IMGC936, an ADC that targets ADAM9, a cell surface protein over-expressed in various solid tumor types. Further, the company develops MGD014 and MGD020, a DART molecule to target the envelope protein of human immunodeficiency virus infected cells and CD3 on T cells; Teplizumab for the treatment of type 1 diabetes; and PRV-3279, a CD32B × CD79B DART molecule for the treatment of autoimmune indications. It has collaborations with Incyte Corporation; Zai Lab Limited; I-Mab Biopharma; and Janssen Biotech, Inc. The company was incorporated in 2000 and is headquartered in Rockville, 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.