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Calliditas Therapeutics AB (publ), a commercial-stage specialty pharmaceutical company, focused on identifying, developing, and commercializing pharmaceuticals products for treatments in orphan indications with initial focus on renal and hepatic diseases. It offers Nefecon, an oral formulation of budesonide that is an immunosuppressant for the treatment of the autoimmune renal disease immunoglobulin A nephropathy. The company's lead product candidate is Setanaxib, a NOX inhibitor that is in Phase 2b/3 clinical trial for the treatment of primary biliary cholangitis; and in Phase 2 clinical trial for the treatment of squamous cell carcinoma of the head and neck cancer and idiopathic pulmonary fibrosis, as well as for type 1 diabetic kidney disease. It also develops Budenofalk 3 mg oral capsules for the treatment of autoimmune hepatitis. The company was founded in 2004 and is headquartered in Stockholm, Sweden.

Geron Corporation, a late-stage clinical biopharmaceutical company, focuses on the development and commercialization of therapeutics for myeloid hematologic malignancies. It develops imetelstat, a telomerase inhibitor that is in Phase 3 clinical trials, which inhibits the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies for the treatment of low or intermediate-1 risk myelodysplastic syndromes and intermediate-2 or high-risk myelofibrosis. The company was incorporated in 1990 and is headquartered in Foster City, California.

IVERIC bio, Inc., a biopharmaceutical company, focuses on the discovery and development of novel treatments for retinal diseases with unmet medical needs. The company develops Zimura, an inhibitor of complement factor C5 comprising GATHER1, which has completed Phase 2/3 clinical trial; GATHER2 that is in Phase 3 clinical trial for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD); and STAR or OPH2005 that is in Phase 2b clinical trial for the treatment of autosomal recessive Stargardt disease. Its preclinical product candidatures include IC-500, a high temperature requirement A serine peptidase 1 protein inhibitors for the treatment of GA and other age-related retinal diseases. The company also develops OPH2001 that has completed Phase 1/2a clinical trial of various doses of avacincaptad pegol (ACP) administered as a monotherapy for the treatment of GA; OPH2000, which has completed Phase 1/2a clinical trial of various doses of ACP administered in combination with Lucentis for the treatment of wet AMD; OPH2007 that has completed Phase 2a clinical trial for the treatment of wet AMD; and OPH2002, which has completed Phase 2a clinical trial for the treatment of idiopathic polypoidal choroidal vasculopathy. In addition, its minigene programs comprises miniCEP290 program for LCA10; miniABCA4 Program for STGD1; and miniUSH2A Program for usher syndrome type 2A-Related inherited retinal diseases (IRDs). The company was formerly known as Ophthotech Corporation and changed its name to IVERIC bio, Inc. in April 2019. The company was incorporated in 2007 and is based in Parsippany, New Jersey. As of July 11, 2023, IVERIC bio, Inc. operates as a subsidiary of Astellas US Holding, Inc.

VBI Vaccines Inc., a biopharmaceutical company, develops and sells vaccines to treat immuno-oncology and infectious disease. It offers Sci-B-Vac, a prophylactic hepatitis B (HBV) vaccine. The company also develops VBI-2601, an immunotherapeutic candidate for the treatment of chronic HBV infection; VBI-1901, a glioblastoma vaccine immunotherapeutic candidate, which is in Phase I/IIa clinical study to treat solid tumors; VBI-1501, a prophylactic cytomegalovirus vaccine candidate that has completed Phase I clinical trial; and VBI-2501 that is in preclinical trial to treat Zika virus. In addition, it develops coronavirus vaccine candidates that include VBI-2902, VBI-2901, and VBI-2905. The company has collaboration and license agreements with Brii Biosciences Limited; GlaxoSmithKline Biologicals S.A.; and the National Research Council of Canada to develop pan-coronavirus vaccine candidate targeting COVID-19, severe acute respiratory syndrome, and Middle East respiratory syndrome. It also has a collaboration with Coalition for Epidemic Preparedness Innovations to advance vaccine candidates against Covid-19 variants. The company was formerly known as SciVac Therapeutics Inc. and changed its name to VBI Vaccines Inc. in May 2016. VBI Vaccines Inc. is based in Cambridge, Massachusetts.

Vanda Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of therapies to address high unmet medical needs worldwide. The company's marketed products include HETLIOZ to treat non-24-hour sleep-wake disorders; and Fanapt oral tablets for the treatment of schizophrenia. Its pipeline products include HETLIOZ (tasimelteon) to treat jet lag disorder, insomnia, delayed sleep phase disorder, sleep disturbances in autism spectrum disorder, and pediatric Non-24; Fanapt (iloperidone) for the treatment of bipolar I disorder and Parkinson's disease psychosis, as well as a long acting injectable (LAI) formulation to treat schizophrenia; and Tradipitant (VLY-686), a small molecule neurokinin-1 receptor (NK-1R) antagonist to treat gastroparesis, motion sickness, atopic dermatitis, and COVID-19 pneumonia. The company's pipeline products also include VTR-297, a small molecule histone deacetylase inhibitor to treat hematologic malignancies and with potential use as a treatment for various oncology indications; VQW-765, a small molecule nicotinic acetylcholine receptor partial agonist to treat performance anxiety and psychiatric disorders; VHX-896, an active metabolite of iloperidone; and antisense oligonucleotide molecules. In addition, it offers a portfolio of cystic fibrosis transmembrane conductance regulator activators and inhibitors that include VSJ-110 for the treatment of dry eye and ocular inflammation; and VPO-227 for the treatment of secretory diarrhea disorders comprising cholera. Vanda Pharmaceuticals Inc. was incorporated in 2002 and is headquartered in Washington, the District of Columbia.