Corcept Therapeutics
NASDAQ:CORTCorcept Therapeutics Incorporated engages in discovery and development of drugs for the treatment of severe endocrinologic, oncologic, metabolic, and neurologic disorders in the United States. It offers Korlym tablets medication for the treatment of hyperglycemia secondary to hypercortisolism in adult patients with endogenous cushing's syndrome; and who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery. The company is also developing relacorilant, which is in phase III clinical trial for the treatment of cushing's syndrome; treatment for adrenal cancer and cortisol excess which is in phase 1b clinical trial; treatment for prostate cancer which is in phase II clinical trial; and nab-paclitaxel in combination with relacorilant, which is in phase III clinical trial to treat platinum-resistant ovarian tumors. In addition, it develops dazucorilant, which is in phase II clinical trial for the treatment of amyotrophic lateral sclerosis; miricorilant, which is in phase IIb trial for the treatment of nonalcoholic steatohepatitis; and treatment for antipsychotic induced weight gain that is in phase I trial. The company was incorporated in 1998 and is headquartered in Menlo Park, California.
Cryoport
NASDAQ:CYRXCryoport, Inc. provides temperature-controlled supply chain solutions in biopharma/pharma, animal health, and human reproductive medicine markets in the Americas, Europe, the Middle East, Africa, and the Asia Pacific. The company offers Cryoport Express Shippers; Cryoport ELITE -80°C Gene Therapy Shipper; Cryoport ELITE Cryosphere Shipper; Cryoport consulting services, including physical, thermal, and shipping qualifications of shipping systems and/or packaging to developing custom packaging solutions; and Cryoport bioservices, such as controlled temperature storage, fulfilment, kit production, secondary packaging, labelling of therapeutic products, and GMP raw materials storage services. It also provides CRYOGENE, an unparalleled solution that offers pre-clinical temperature-controlled biological materials management services comprising specimen storage, processing, collection, and retrieval; CRYOPD, a temperature-controlled logistics solution, which include temperature-controlled packaging and transport solutions from cryogenic temperature to controlled ambient; and IntegriCell services that comprise apheresis/leukapheresis collection, Cryoshuttle transportation, cryo-process optimization and processing services. In addition, the company offers MVE Biological Solutions' Fusion cryogenic system, a self-sustaining cryogenic freezer; MVE Biological Solutions' Vario cryogenic system, a cryogenic freezer system that supports temperatures between -20°C and -150°C; Cryoport Cryoshuttle provides clients with dedicated local transportation support; and Tec4Med. Further, it provides biological specimen cryopreservation storage and maintenance; archiving, monitoring, tracking, receipt, and delivery of samples; frozen biological specimens transportation; and incoming and outgoing biological specimens management; and short-term logistics and engineering consulting services. Cryoport, Inc. was founded in 1999 and is headquartered in Brentwood, Tennessee.
Dicerna Pharmaceuticals
NASDAQ:DRNADicerna Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to selectively silence genes that cause or contribute to disease. The firm uses its proprietary GalXC RNAi technology platform to build a pipeline in these therapeutic areas. The company was founded by Douglas M. Fambrough III, Mark A. Behlke, Roberto Guerciolini, James Craig Jenson, and John J. Rossi in October 2006 and is headquartered in Lexington, MA.
Amicus Therapeutics
NASDAQ:FOLDAmicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late-onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. The company was incorporated in 2002 and is headquartered in Princeton, New Jersey.
Rocket Pharmaceuticals
NASDAQ:RCKTRocket 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.