Eiger BioPharmaceuticals
NASDAQ:EIGREiger BioPharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of targeted therapies for rare and ultra-rare diseases in the United States and internationally. Its lead product candidate is Lonafarnib, an orally bioavailable, small molecule, which is in Phase III clinical trials to treat hepatitis delta virus infection. The company's product candidate also include Lambda, which targets type III interferon receptors that has completed Phase II clinical trials; Lonafarnib for the treatment of progeria and progeroid laminopathies; and Avexitide for the treatment of congenital hyperinsulinism, as well as has completed Phase II clinical trials to treat post-bariatric hypoglycemia. Eiger BioPharmaceuticals, Inc. was founded in 2008 and is headquartered in Palo Alto, California.
Gamida Cell
NASDAQ:GMDAGamida Cell Ltd., a clinical-stage biopharmaceutical company, develops cell therapies to cure blood cancers and serious blood diseases. The company's lead product candidate is omidubicel, a cell therapy that has completed Phase III clinical trial in patients with hematologic malignancies, as well as in Phase II clinical trials in patients with severe aplastic anemia. It is also developing GDA-201, an investigational NK cell-based cancer immunotherapy, which is in Phase I/II studies for the treatment of relapsed or refractory non-Hodgkin lymphoma and multiple myeloma. The company was incorporated in 1998 and is headquartered in Jerusalem, Israel.
Oncobiologics
NASDAQ:ONSOutlook Therapeutics, Inc. is a clinical-stage biopharmaceutical company, which engages in the identification, development, manufacture, and commercialization of ONS-5010/LYTENAVA as the first FDA-approved ophthalmic formulation of bevacizumab-vikg for use in retinal indications, including wet AMD, DME and BRVO. The company was founded by Pankaj Mohan on January 5, 2010 and is headquartered in Iselin, NJ.
Solid Biosciences
NASDAQ:SLDBSolid Biosciences Inc., a life science company, engages in developing therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a next-generation gene transfer candidate for the treatment of Duchenne muscular dystrophy (Duchenne); AVB-202-TT, a gene therapy program for the treatment of Friedreich's ataxia; AVB-401, a gene therapy program for the treatment of BAG3-mediated dilated cardiomyopathy; and other drugs for the treatment of undisclosed cardiac diseases. It also engages in developing platform technologies, including dual gene expression, a technology for packaging multiple transgenes into one vector, as well as novel capsids. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.
X4 Pharmaceuticals
NASDAQ:XFORX4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases. Its lead product candidate is mavorixafor, a small molecule inhibitor of the chemokine receptor C-X-C chemokine receptor type 4 (CXCR4), which is in Phase III clinical trial for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome; and Phase II clinical trial to treat congenital, idiopathic, or cyclic neutropenia. The company is also developing X4P-003, a CXCR4 antagonist for the treatment of CXCR4 dependent disorders and primary immunodeficiencies; and X4P-002, a CXCR4 antagonist for the treatment of blood-brain barrier diseases. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications. The company is headquartered in Boston, Massachusetts.