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.
Madrigal Pharmaceuticals
NASDAQ:MDGLMadrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development of therapeutics for the treatment of non-alcoholic steatohepatitis (NASH) in the United States. Its lead product candidate is resmetirom, a liver-directed thyroid hormone receptor beta agonist, which is in Phase 3 clinical trials for treating NASH. The company is headquartered in West Conshohocken, Pennsylvania.
Mersana Therapeutics
NASDAQ:MRSNMersana Therapeutics, Inc., a clinical stage biopharmaceutical company, develops antibody drug conjugates (ADC) for cancer patients with unmet needs. The company develops XMT-1660, a B7-H4-targeted Dolasynthen ADC candidate; and XMT-2056, an immunosynthen ADC. It has research and development collaborations with Janssen Biotech, Inc., Ares Trading S.A., Merck KGaA, and Asana BioSciences, LLC for the development of ADC product candidates. The company was formerly known as Nanopharma Corp. and changed its name to Mersana Therapeutics, Inc. in November 2005. Mersana Therapeutics, Inc. was incorporated in 2001 and is headquartered in Cambridge, Massachusetts.
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.
Trillium Therapeutics
NASDAQ:TRILTrillium Therapeutics, Inc. is a clinical stage immuno-oncology company, which engages in the research and development of therapies for the treatment of cancer. It offers clinical programs, TTI-621 and TTI-622, target CD47, a signal that cancer cells frequently use to evade the immune system. The company was founded on March 31, 2004 and is headquartered in Mississauga, Canada.