(US) Aerie Pharmaceuticals, Inc. engages in the discovery, development, and commercialization of therapies for the treatment of patients with open-angle glaucoma, retinal diseases and other diseases of the eye. Its products include Rhopressa, Roclatan, AR-13503, and AR-1105. The company was founded by David L. Epstein, Casey C. Kopczynski, Thomas J. van Haarlem, and Eric J. Toone on June 22, 2005 and is headquartered in Durham, NC.
IVERIC bio, Inc. is a biopharmaceutical company which engages in discovering, developing, and commercializing novel therapeutics to treat ophthalmic diseases, with a focus on age-related and orphan retinal diseases. Its product candidates include Zimura and Gene Therapy. The company was founded by David R. Guyer and Samir Chandrakant Patel on January 5, 2007 and is headquartered in New York, NY.
(US) Epizyme, Inc. is a clinical-stage biopharmaceutical company. The firm engages in the discovery, development, and commercialization of novel epigenetic medicines for cancer and other diseases. Its product pipeline is lead by, tazemetostat which targets Non-Hodgkin Lymphoma, molecularly defined solid tumors, non-small-cell lung carcinoma, molecularly targeted tumors, and ovarian cancer. The firm also develops pinometostat for genetically defined acute leukemia, and EZM8266 for sickle cell disease. The company was founded by Robert Horvitz and Zhang Yi on November 1, 2007 and is headquartered in Cambridge, MA.
(US) NanoString Technologies, Inc. engages in the development and commercialization of instrument and services for profiling of genes and proteins from tissue sample. It offers GeoMx Digital Spatial Profiler, nCounter Systems, analysis solutions, gene expression panels, Vantage 3D Assays, miRNA Assays, custom solutions, and Sample Prep and nCounter Consumables. The company was founded by Amber Ratcliffe, Krassen Dimitrov, and Dwayne Dunaway on June 20, 2003 and is headquartered in Seattle, WA.
(US) Agios Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in the discovery and development of novel investigational medicines to treat cancer and rare genetic diseases. It focuses on diseases that are directly caused by changes in genes or chromosomes, often passed from one generation to the next. The company was founded by Lewis Clayton Cantley, Tak W. Mak, Craig B. Thompson and Shin-Shan Michael Su on August 7, 2007 and is headquartered in Cambridge, MA.
Arcturus Therapeutics Holdings, Inc. engages in the research and development of medical applications for the nucleic acid-focused technology. The firm develops RNA therapeutics which focuses on the treatment of liver and respiratory diseases. Its pipeline include LUNAR-OTC and LUNAR CF. The company was founded in 2013 and is headquartered in San Diego, CA.
bluebird bio, Inc. is a clinical-stage biotechnology company, which engages in the development of potential gene therapies for severe genetic diseases and cancer. Its integrated product platform includes gene therapy, cancer immunotherapy, and gene editing. Its product pipeline includes Lenti-D, LentiGlobin, BCL11a shRNA, bb2121, and bb21217. The company was founded by Philippe Leboulch and Ronald C. Dorazio on April 16, 1992 and is headquartered in Cambridge, MA.
(US) Chimerix, Inc. is a biopharmaceutical company, which engages in the research, development, and commercialization of pharmaceutical products. Its portfolio includes Brincidofovir for the treatment of adenovirus, cytomegalovirus, and smallpox. The company was founded by George R. Painter, III and Timothy Wollaeger in April 2000 and is headquartered in Durham, NC.
(US) Enanta Pharmaceuticals, Inc. operates as a biotechnology company. It engages in the research and development of molecule drugs for the treatment of viral infections and liver diseases. The firm offers the medicine under the brands Mavyret and Viekira Pak. The company was founded by Peter O. Kliem and Gregory L. Verdine in 1995 and is headquartered in Watertown, MA.
(US) Esperion Therapeutics, Inc. engages in the development and commercialization of oral therapies for the treatment of patients with elevated low-density lipoprotein cholesterol. It offers NEXLETOL (bempedoic acid) tablets, and NEXLIZET (bempedoic acid and ezetimibe) tablets. The company was founded by Roger S. Newton, Hans Ageland, Jan O. Johansson, Anders Paul Wiklund, Michael E. Pape, David I. Scheer and Charles L. Bisgaier in May 1998 and is headquartered in Ann Arbor, MI.
Evoke Pharma, Inc. is a pharmaceutical company, which engages in the development of drugs for the treatment of gastrointestinal disorders and diseases. It develops Gimoti, with promotility and anti-emetic effects, for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in women with diabetes mellitus. The company was founded by Matthew J. D'Onofrio, Cam L. Garner, Scott L. Glenn, and David A. Gonyer in January 2007 and is headquartered in Solana Beach, CA.
(US) Fate Therapeutics, Inc. engages in the development of programmed cellular immunotherapies for cancer and immune disorder. Its pipeline of products include immuno-ocology candidates and immuno-regulation candidates. The company was founded by Philip Beachy, Sheng Ding, Rudolf Jaenisch, Randall T. Moon, Michael Rudnicki, David Scadden, Leonard Zon, Alexander Rives, Scott Wolchko, and John D. Mendlein on April 27, 2007 and is headquartered in San Diego, CA.
Histogen, Inc. engages in the development of patented, innovative technologies that replace and regenerate tissues in the body for the aesthetic and therapeutic markets. Its technology is based on the discovery that growing fibroblast cells under simulated embryonic conditions induces them to become multipotent stem cells. The environment created by its proprietary process mimics the conditions within the womb very low oxygen and suspension. When cultured under these conditions, the induced multipotent stem cells generate biological materials that stimulate a patient’s own stem cells to activate and regenerate tissue. This results in potential therapeutic benefits in multiple treatment settings, including skin care, orthopedic indications such as cartilage formation and spinal disc repair, wound healing and dermal fillers. The company was founded on July 13, 2005 by Steven J. Mento and is headquartered in San Diego, CA.
Kamada Ltd. engages in the development and production of pharmaceutical products. It operates through the Proprietary Products segment and the Distribution segment. The Proprietary Products segment covers the development, manufacture, and sale of plasma-derived protein therapeutics products. The Distribution segment covers the supply of plasma-based products for clinical use. The company was founded by David Tsur and Ralf Hahn on December 13, 1990 and is headquartered in Rehovot, Israel.
(US) Karyopharm Therapeutics, Inc. is an oncology-focused pharmaceutical company dedicated to the discovery, development, and commercialization of novel first-in-class drugs directed against nuclear export and related targets for the treatment of cancer and other major diseases. Its SINE compounds function by binding with and inhibiting the nuclear export protein XPO1 (or CRM1). Karyopharm’s lead compound, XPOVIOTM (selinexor), received accelerated approval from the FDA in combination with dexamethasone as a treatment for patients with heavily pretreated multiple myeloma. A Marketing Authorization Application for selinexor is also currently under review by the European Medicines Agency (EMA). In addition to single-agent and combination activity against a variety of human cancers, SINE compounds have also shown biological activity in models of neurodegeneration, inflammation, autoimmune disease, certain viruses and wound-healing. Karyopharm has several investigational programs in clinical or preclinical development. The company was founded by Joseph Araujo, Ronald A. DePinho, Pamela A Silver, Giulio Draetta, Michael G. Kauffman and Sharon Shacham on December 22, 2008 and is headquartered in Newton, MA.
(US) MacroGenics, Inc. is a clinical-stage biopharmaceutical company, which engages in discovering and developing antibody-based therapeutics designed to modulate the human immune response for the treatment of cancer. 2. Its product pipeline includes Margetuximab, Flotetuzumab, Enoblituzumab, MGA012, MGD013, MGD019, MGD009, MGC018, and MGD007 for oncology; and MGD014 for infectious diseases. The company was founded by Scott E. Koenig, Jeffrey V. Ravetch, LeRoy E. Hood, Ruedi Aebersold, and Alan Aderem on August 14, 2000 and is headquartered in Rockville, MD.
(US) Onconova Therapeutics, Inc. is a clinical-stage biopharmaceutical company, which engages in the identification and development of oncology therapeutics. It focuses on discovering and developing small molecule drug candidates to treat cancer. The company was founded by Ramesh Kumar and E. Premkumar Reddy on December 22, 1998 and is headquartered in Newtown, PA.
(US) PTC Therapeutics, Inc. is a biopharmaceutical company, which engages in the discovery and commercialization of clinically-differentiated medicines. It focuses on the development of new treatments for multiple therapeutic areas, including rare diseases and oncology. The company was founded by Allan Steven Jacobson and Stuart W. Peltz on March 31, 1998 and is headquartered in South Plainfield, NJ.
(US) Tandem Diabetes Care, Inc. engages in the design, development, and commercialization of products for people with insulin-dependent diabetes. Its flagship product, t:slim X2 Insulin Delivery System, operates as a small insulin pump. The company was founded by Paul M. DiPerna on January 27, 2006 and is headquartered in San Diego, CA.
(US) Veracyte, Inc. engages in the research, development and commercialization of diagnostic products. The firm's portfolio includes Afirma, Percepta, and Envisia. It intends to treat thyroid cancer, improve lung cancer screening, and clarify the diagnosis of idiopathic pulmonary fibrosis. The company was founded by Bonnie H. Anderson and Y. Douglas Dolginow on August 15, 2006 and is headquartered in South San Francisco, CA.