ADMA Biologics
NASDAQ:ADMAADMA Biologics, Inc., a biopharmaceutical company, engages in developing, manufacturing, and marketing specialty plasma-derived biologics for the treatment of immune deficiencies and infectious diseases in the United States and internationally. The company offers BIVIGAM, an intravenous immune globulin (IVIG) product indicated for the treatment of primary humoral immunodeficiency (PI); ASCENIV, an IVIG product for the treatment of PI; and Nabi-HB for the treatment of acute exposure to blood containing Hepatitis B surface antigen and other listed exposures to Hepatitis B. It develops a pipeline of plasma-derived therapeutics, including products related to the methods of treatment and prevention of S. pneumonia infection for an immunoglobulin. In addition, it operates source plasma collection facilities. The company sells its products through independent distributors, drug wholesalers, specialty pharmacies, and other alternate site providers. ADMA Biologics, Inc. was incorporated in 2004 and is headquartered in Ramsey, New Jersey.
Applied Genetic Technologies
NASDAQ:AGTCApplied Genetic Technologies Corporation, a clinical-stage biotechnology company, develops transformational genetic therapies for patients suffering from rare and debilitating diseases. Its advanced product candidates include three ophthalmology development programs across two targets, including X-linked retinitis pigmentosa, which is in the Phase 1/2 clinical trials; and achromatopsia that is in Phase 1/2 clinical trials. It is also developing an optogenetic product candidate to treat advanced retinal disease. In addition, the company has initiated one preclinical program in otology; preclinical program in dry age-related macular degeneration; and two preclinical programs in targeting central nervous system disorders, including frontotemporal dementia and amyotrophic lateral sclerosis. It has collaboration agreements with University of Florida; Bionic Sight, Inc.; and Otonomy, Inc. The company was incorporated in 1999 and is headquartered in Alachua, Florida.
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.
Genfit
NASDAQ:GNFTGenfit S.A., a biopharmaceutical company, discovers and develops drug candidates and diagnostic solutions for metabolic and liver-related diseases. The company develops Elafibranor, which is in Phase III clinical trial to treat patients with primary biliary cholangitis. It also engages in the development of NIS4 technology for the diagnosis of nonalcoholic steatohepatitis (NASH) and fibrosis; VS-01 for the treatment of Urea Cycle Disorder (UCD) and Organic Acidemia Disorder (OAD); GNS561, which is in Phase 1b/2 trial to treat patients with cholangiocarcinoma (CCA); and VS-01-ACLF and Nitazoxanide (NTZ), which is in Phase 1 trial to treat acute-on-chronic liver failure, as well as VS-02-HE, which is in preclinical trial for the treatment of Reduction of Hyperammonemia and the Stabilization of Blood Ammonia. The company has a licensing agreement with Labcorp for the commercialization of NASHnext, a blood-based molecular diagnostic test; and Genoscience Pharma to develop and commercialize the investigational treatment GNS561 for CCA. The company was incorporated in 1999 and is headquartered in Loos, France.
LogicBio Therapeutics
NASDAQ:LOGCLogicBio Therapeutics, Inc., a genetic medicine company, focuses on developing and commercializing genome editing and gene therapy treatments using its GeneRide and sAAVy platforms. The company's GeneRide technology is a new approach to precise gene insertion harnessing a cell's natural deoxyribonucleic acid; and gene delivery platform, sAAVy is an adeno-associated virus, which is designed to optimize gene delivery for treatments in a range of indications and tissues. Its lead product candidate is LB-001 that is in Phase I/II clinical trials for the treatment of methylmalonic acidemia. The company has a collaboration with Children's Medical Research Institute to develop next-generation capsids for gene therapy and gene editing applications in the liver, as well as additional tissues; and a collaboration agreement with Takeda Pharmaceutical Company Limited to develop LB-301, an investigational therapy for the treatment of Crigler-Najjar syndrome. The company also has a research collaboration, license, and option agreement with CANbridge Care Pharma Hong Kong Limited; and collaboration agreement with Daiichi Sankyo Company. The company was incorporated in 2014 and is headquartered in Lexington, Massachusetts.